Podcasts about trikafta

"Many people have their own thing that lights them on fire, for me, it's running" say Katie O'Grady.  CF modulator drugs changes are a game changer for people living with cystic fibrosis. Katie O'Grady's story is a powerful reflection of how dramatically life with cystic fibrosis has changed in the era of CF modulators, and what becomes possible when hope replaces survival mode. A runner, speaker, and cystic fibrosis advocate in Boston, Katie redefined what it means to live with CF. Katie inspires us by talked about her shift in mindset and how it changed everything: she stopped thinking of herself as “a CF patient who runs” and began identifying as “a runner who happens to have CF.” That subtle but profound change transformed not only the way she trained, but the way she cared for herself, viewed her future, and moved through the world. For much of her life, Katie lived the reality many people with CF know too well — moving from infection to infection, never fully certain what the next year, or even the next season, might bring. At 18, she battled a devastating case of pneumonia that forced her to confront the fragility of her health. But in 2019, everything changed with the arrival of Trikafta. For the first time, Katie could imagine a future without immediately attaching fear or limitations to it. She speaks candidly about what it means to rebuild trust in your body after years of illness, and how running became far more than exercise. It became freedom, therapy, identity, and proof that her diagnosis did not get to define the boundaries of her life. Katie opens up about taking the longest break from running she'd ever experienced, wondering if she would ever return, and discovering that what she missed most wasn't competition — it was the clarity, peace, and sense of self she found while moving. To watch on YouTube: https://www.youtube.com/watch?v=zzdQraUAPfc Like, subscribe, and comment on our podcasts!Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website:https://thebonnellfoundation.orgEmail us at: thebonnellfoundation@gmail.com Watch our podcasts on YouTube: https://www.youtube.com/@laurabonnell1136/featuredNew: Shop our merchandise! https://thebonnellfoundation.org/product-shop/Thanks to our sponsors:Vertex: https://www.vrtx.comViatris: https://www.viatris.com/enRead us on Substack: https://substack.com/@lstb?utm_campaign=profile&utm_medium=profile-pageWatch our trailer of Embracing Egypt: https://youtu.be/RYjlB25Cr9Y

This week on Sickboy we bring you the first episode of the latest season of Breathless. We're proud as heck with how this panned out and feel like you will dig it! The Season 2 premiere of Breathless explores a biological and existential revolution. For decades, Cystic Fibrosis was defined by a "physical perimeter"—a list of things that were simply impossible. But with the advent of transformative drugs like Trikafta, those walls are vanishing. Host Jeremie opens with a visceral comparison between a 2017 spiritual "holotropic breathing" experience and the 2021 pharmaceutical miracle of his first dose of Trikafta. We then hear from elite athletes who shattered expectations long before the "miracle drug" existed, and we look at how Cystic Fibrosis Canada is pivoting its entire mission from "extending life" to supporting a life "without limits."

This week on Sickboy we bring you the first episode of the latest season of Breathless. We're proud as heck with how this panned out and feel like you will dig it! The Season 2 premiere of Breathless explores a biological and existential revolution. For decades, Cystic Fibrosis was defined by a "physical perimeter"—a list of things that were simply impossible. But with the advent of transformative drugs like Trikafta, those walls are vanishing. Host Jeremie opens with a visceral comparison between a 2017 spiritual "holotropic breathing" experience and the 2021 pharmaceutical miracle of his first dose of Trikafta. We then hear from elite athletes who shattered expectations long before the "miracle drug" existed, and we look at how Cystic Fibrosis Canada is pivoting its entire mission from "extending life" to supporting a life "without limits."

Episode SummaryIn the Season 2 finale of Breathless, host Jeremie Saunders tackles the ultimate question facing the Cystic Fibrosis (CF) community: Are we finally on the verge of a true cure? Growing up sick, Jeremie developed a psychological "immunity" to the repetitive promise that a cure was "just around the corner." But today, the conversation is fundamentally different.This episode takes us out of the pharmacy and directly into the laboratories where researchers are transitioning from small-molecule symptom management to structural gene editing. Featuring Dr. Bowen Li (University of Toronto), Dr. Paul Eckford (Chief Scientific Officer, CF Canada), and Kelly Grover (CEO, CF Canada), we pull back the curtain on the grueling reality of scientific research—described as "pushing a boulder up a hill with chopsticks." We explore how the global proof of concept from COVID-19 mRNA vaccines has turbocharged CF research, how CRISPR molecular scissors are being engineered to permanently rewrite genetic typos, and how Machine Learning is drastically accelerating the timeline to reach the final 10% of the community.Main Topics & Key Moments1. The Cost of Hope & The Ground ShiftThe Practiced Optimism: Jeremie details the emotional exhaustion of childhood hope cycles, where promised breakthroughs consistently failed to materialize, leading to a defensive "self-preservation" mindset in his twenties.A New Vocabulary: Why current scientific progress is materially different. For the first time, data-driven researchers are using definitive terms like functional cure, mutation-agnostic, and restoring normal function at the source.2. The Grind of the ArchitectureChopsticking the Boulder: Dr. Bowen Li describes the quiet, unglamorous reality of the lab at 8:00 PM on a Tuesday, where progress is measured in microscopic, hard-won inches.The Lazarus Effect vs. The Temped Joy: CEO Kelly Grover recalls an early advocacy trip to Ottawa with a profoundly ill young man, contrasting his miraculous post-Trikafta transformation with the sobering reality of meeting patients who remain devastatingly sick.3. The Molecular Typo: Nonsense MutationsThe Broken Text: While Trikafta acts as a structural prop for a wobbly, misfolded protein, it is entirely useless for patients with "nonsense mutations."The Cellular Stop Sign: Dr. Bowen Li explains that in these rare variations (such as the W57X mutation discussed in previous episodes), the cell hits a premature stop sign in the middle of reading the genetic sentence. The protein is cut off too early and never forms, leaving nothing for modulator drugs to attach to.4. Photocopies and Vaults: mRNA vs. CRISPRThe Vault Analogy: Dr. Li frames human genetics simply: DNA is the master cookbook safely locked inside the nucleus vault. You cannot remove the book, but you can create a temporary photocopy of a single page to take to the kitchen. That photocopy is mRNA.The Platform Revolution: A historical look at mRNA, from its discovery by Brenner and Crick in the 1960s to Katalin Karikó's underfunded, decades-long battle to chemically stabilize the molecule. The global deployment of COVID-19 vaccines served as the ultimate medical proof of concept, opening the floodgates for respiratory disease mapping.The Lipid Nanoparticle Trojan Horse: To get delicate mRNA past the lung's natural "bouncers" (mucus and cilia), scientists wrap it in a micro-engineered fat bubble designed to slide through sticky blockages and hit targeted cells.CRISPR Molecular Scissors: Moving beyond temporary photocopies, CRISPR edits the master cookbook itself. Hijacked from a bacterial immune defense system discovered in yogurt cultures, Jennifer Doudna and Emmanuelle Charpentier engineered the Cas9 protein to act as scissors guided by genetic GPS. Modern iterations—like base editing and prime editing—can cleanly correct a single letter in the double helix without fracturing the strand. Hosted on Acast. See acast.com/privacy for more information.

What do you do when a life-saving medical breakthrough saves almost everyone in your chronic illness community—except you? If you're new to the show, Cystic Fibrosis is traditionally known as a genetic disease that heavily impacts the lungs. But this week, the fellas sit down with Nicholas Kelly, a registered dietitian who playfully calls himself a "unicorn" because he's a Black man living with a condition stereotypically labeled as a pasty white person's illness. (Oh, and Taylor passed out right before we hit record, so he's currently sleeping off-set while Brian stands by with his historically rib-shattering CPR skills) . Nick opens up about his mother bucking the 1980s medical establishment to diagnose him at three months old , outliving a massive string of childhood expiration dates , and surviving an actual "code blue" post-surgery nightmare. He also drops a beautifully perspective-shifting bomb on the room about what it truly feels like to watch his closest friends get saved by the miracle drug Trikafta while his own rare genetic mutations leave him completely ineligible for the club. From highly inappropriate anatomy questions to a profound psychological transition from chasing the clock to building a legacy , this episode is an incredible look at what it means to live life with absolute passion.Follow Sickboy: Instagram: https://www.instagram.com/sickboypodcastTiktok: https://www.tiktok.com/@sickboypodcastDiscord: https://discord.gg/expeUDN

What do you do when a life-saving medical breakthrough saves almost everyone in your chronic illness community—except you? If you're new to the show, Cystic Fibrosis is traditionally known as a genetic disease that heavily impacts the lungs. But this week, the fellas sit down with Nicholas Kelly, a registered dietitian who playfully calls himself a "unicorn" because he's a Black man living with a condition stereotypically labeled as a pasty white person's illness. (Oh, and Taylor passed out right before we hit record, so he's currently sleeping off-set while Brian stands by with his historically rib-shattering CPR skills) . Nick opens up about his mother bucking the 1980s medical establishment to diagnose him at three months old , outliving a massive string of childhood expiration dates , and surviving an actual "code blue" post-surgery nightmare. He also drops a beautifully perspective-shifting bomb on the room about what it truly feels like to watch his closest friends get saved by the miracle drug Trikafta while his own rare genetic mutations leave him completely ineligible for the club. From highly inappropriate anatomy questions to a profound psychological transition from chasing the clock to building a legacy , this episode is an incredible look at what it means to live life with absolute passion.Follow Sickboy: Instagram: https://www.instagram.com/sickboypodcastTiktok: https://www.tiktok.com/@sickboypodcastDiscord: https://discord.gg/expeUDN

Episode SummaryWhat happens when the parade moves on, but you're still standing on the sidewalk? In this heavy and deeply candid episode of Breathless, host Jeremie Saunders addresses the modern paradox of the "Trikafta Revolution." While a miraculous 90% of the Cystic Fibrosis community is experiencing a historical second chance at life, a remaining 10% is left stranded in the old world.We follow the story of Teresa Weger, a 20-year-old university student from Weyburn, Saskatchewan. Teresa possesses an ultra-rare, spontaneous genetic mutation that places her entirely outside the reach of current modulator drugs. Jeremie pairs Teresa's current physical decline with his own history of severe CF complications, confronting the painful reality of survivor's guilt. This episode pulls back the curtain on the emotional isolation of being left behind by your own community, while exploring the cutting-edge genetics and future technologies—like mRNA and CRISPR—needed to finish the fight for everyone.Main Topics & Key Moments1. The Meshed Reality of a RevolutionThe Shrinking Base: Teresa Weger notes a drastic shift in the CF community. As health dramatically improves for the majority, community enthusiasm and attendance at advocacy events (like local walks) have noticeably waned.Tempered Joy: CF Canada CEO Kelly Grover notes that while reaching "first base" is a biological triumph, the organization's celebrations must remain tempered until therapeutic options exist for 100% of patients.2. The Genetic Lottery & Spontaneous MutationThe "Toyota Camry" vs. The Custom Model: The vast majority of CF patients carry the common Delta F508 mutation. Teresa's mutation is an ultra-rare, complex anomaly: W57X/exon 23-24 deletion.The Biological Glitch: Chief Scientific Officer Dr. Paul Eckford explains a rare break from the classic inheritance model. While Teresa's mother is a standard carrier, her father is not. Teresa's CF was caused by a completely spontaneous, random genetic mutation during DNA replication at the exact moment of conception.3. The Physical Toll of the Old WorldThe Linear Decline: After maintaining a stable childhood with a lung function of 97%, Teresa experienced a sudden adolescent crash, with her lung capacity dropping into the 50s.The Multi-Front War: A graphic look at severe CF symptoms outside the lungs, highlighting Teresa's struggles with chronic gastrointestinal blockages and painful sinus surgeries that result in post-sedation complications.4. Isolation at the ParadeThe Regina CF Walk: Teresa recalls the painful emotional whiplash of standing backstage at a fundraising walk. Ahead of her, a Trikafta-responsive speaker joyfully proclaimed that her sweat chloride levels had normalized to healthy standards—leaving Teresa to follow with a speech detailing her rapid physical decline.Parallel Tracks, Forked Roads: Jeremie recognizes his own past medical trauma in Teresa's current daily fight—specifically their shared history of aggressive sinus polyps and major large intestine resections 75% of Jeremie's large bowel was removed). He struggles to navigate the raw survivor's guilt of receiving a "golden ticket" while others remain waiting.Key Quotes"The experience of feeling like you're on the same team but no longer working for the same goal... we all still have CF, but they don't still have the same CF as me." — Teresa Weger"It's like winning the lottery, only the prize is a life-limiting genetic disease that no pharmaceutical company has built a drug for because you might be the only person on earth with your specific genetic code." — Host (Jeremie)"I am the one who got to keep living. And she is the one who is still waiting." — Host (Jeremie)"We aren't done. And we won't be done until the bus stops for everyone." — Host (Jeremie) Hosted on Acast. See acast.com/privacy for more information.

Episode SummaryFor parents, caregivers, and anyone living close to cystic fibrosis, this episode asks a painful, hopeful question: what does a CF diagnosis mean for a child born today?Jeremie Saunders follows two families separated by a generation of science. One entered the world of CF before modulators, when a diagnosis landed like grief and the future felt brutally narrow. The other is raising a child diagnosed through newborn screening in the age of Trikafta, where the prognosis conversation has changed—but fear hasn't disappeared.Along the way, we trace the sweat test, newborn screening, pediatric CF care, and the strange emotional terrain of parenting in an era of real medical progress. You'll hear how Trikafta is reshaping childhood, what still keeps parents up at night, and why hope in cystic fibrosis is wider now—but not yet complete.This is an episode about the next generation of CF: born into more possibility, still living with uncertainty.Main Topics & Key Moments1. The 1989 Baseline & The Long CorridorA Strange Year for Hope: Jeremie reflects on his own diagnosis in 1989—the same year scientists discovered the CFTR gene, sparking a wave of hope that evaporated when an immediate cure failed to materialize.Asthma or Something Sinister?: Ross Drake recalls the exhausting years spent treating his youngest son, Ryan, for what they thought was asthma, only to watch him cough until he threw up phlegm every night.The Elevator Ride: Ross shares the heartbreaking moment he received the call confirming his second son, Stephen, also had CF, and having to leave work to tell his wife.2. The Science of Sweat & ScreeningQuantitative Pilocarpine Iontophoresis: A deep dive into the classic CF "sweat test." The CFTR protein acts as a cellular gatekeeper; when it fails, chloride cannot re-enter the cells, making CF children famously "salty to the taste."The Gray Zone: Understanding the diagnostic numbers: under 30 is clear, 30 to 59 is a medical "maybe," and 60 or higher is the definitive threshold where a family's planned future disappears.The Battle for Newborn Screening: How Cystic Fibrosis Canada fought a province-by-province bureaucratic war to mandate infant screening, allowing babies like Miles to be flagged and treated weeks after birth rather than years.3. The Generational DivergenceDiagnosis as Loss vs. Diagnosis Tinted with Hope: While Ross's generation faced a relentless schedule of prevention and a median life expectancy of just 37 years, Brittany was told that while the diagnosis was unfortunate, Miles "couldn't have been born at a better time."Trikafta as a Childhood Landscape: For the new generation, CFTR modulators aren't a mid-life rescue mission; they are the baseline. The drug targets the underlying protein defect, moving the line from symptom management to true physical transformation.4. The Enzyme RevelationBlowing the Medical Mind: Brittany shares a stunning clinical update: because Miles started Trikafta at such a young stage, his pancreas has recovered enough that he has been completely taken off pancreatic enzymes.Preserving the Organ: The medical frontier explores how introducing modulators to infants might prevent chronic organ damage before it ever starts.Key Quotes"Parenting with CF meant building your life around prevention, because medicine couldn't yet offer transformation." — Host (Jeremie)"I got to go home now and tell my wife, my other son has CF." — Ross Drake"As unfortunate as it is that he has been diagnosed with CF, he couldn't have been born at a better time." — Brittany Hollowink"And this has just been within the last three months we've been able to completely take him off his pancreatic enzymes... He's been doing just fine and it's blowing my mind." — Brittany Hollowink Hosted on Acast. See acast.com/privacy for more information.

Episode SummaryIn this powerful episode of Breathless, we explore the boundary between physical restriction and newfound freedom. For decades, Cystic Fibrosis (CF) was a disease defined by limits: the limit of a breath, the limit of a career, and the limit of a lifespan.Today, those limits are being shattered. We follow two extraordinary journeys: Jeremy Vosburgh, a homicide detective who defied medical expectations to serve on the front lines of law enforcement, and Lauren Clift, a musical theater performer who transitioned from "choreographing her coughs" to taking her first full, effortless breath. This episode examines the "CF Without Limits" philosophy—a shift from merely surviving to actively planning for a future that was never guaranteed.Main Topics & Key Moments1. The Performer's MaskThe Hidden Struggle: Lauren Clift describes the grueling reality of performing in Legally Blonde while her lung function declined, forcing her to hide violent coughing fits behind stage choreography.The "Cough Blackout": A reflection on the magic of the stage and how, for years, the adrenaline of performance was the only thing that could temporarily silence the symptoms of CF.2. The Obstacle Course: Jeremy's StoryDefying the System: Jeremy Vosburgh shares how he bypassed the "rules" of CF to become a police officer, proving his capability through a high-intensity fitness test.Pepper Spray & Resilience: The incredible irony of a man with a respiratory disease voluntarily undergoing pepper spray training and physical altercations to protect his community.The 4 AM Discipline: A look at the brutal "old-school" grit required to balance nebs, neubulizers, and a demanding career before modern medicine arrived.3. Left Behind: The Rare Mutation GapThe Trikafta Blackout: While 90% of the CF community celebrated a miracle drug, Lauren was part of the 10% with rare mutations who were "left behind."Genetic Typos: CF Canada's Chief Scientific Officer, Paul Eckford, explains why certain genetic codes don't respond to standard treatments and the isolation that causes for patients.The Wall of Advocacy: How Lauren's mother—a pharmacist—had to beg politicians for the very drug she dispensed to others while watching her own daughter fade.4. "My Precious": The Turning PointThe Miracle Arrives: The bureaucratic and emotional hurdle of finally accessing Trikafta for rare mutations in July 2024.A Full Breath: Lauren describes the profound, tearful moment of rolling over in bed and taking a deep breath without coughing for the first time in years.5. The New FrontierPlanning to be an Old Man: Jeremy reflects on how his goals have shifted from "getting by" to the simple, once-impossible dream of growing old.Beyond the Lungs: CEO Kelly Grover discusses how CF Canada is pivoting to support "best lives"—focusing on fertility, education, and retirement planning.The Weight of the Future: Addressing the survivor's guilt and the mental health challenge of re-entering a world you thought you were leaving.Key Quotes"I would literally have to choreograph into my dance routine where I could cough without the audience hearing... otherwise I wouldn't make it through the show." — Lauren Clift"I don't like being told that it's impossible. I don't mind being told that it might be hard... but I would say that that's [nonsense] and that you can do it." — Jeremy Vosburgh"I dispense this drug to other CF patients that I see getting better. And I am watching my daughter die." — Lauren's Mother"Relief is the biggest thing... but also relief in just breathing. It's really cool to just breathe. I don't know if people know that." — Lauren Clift Hosted on Acast. See acast.com/privacy for more information.

What happens when a miracle drug gives you back your future, but your mind is still built for a shorter life?In this moving episode of Breathless, host Jeremie and guests dive into the "mental health fallout" of growing up with Cystic Fibrosis. For decades, the CF community focused on one goal: survival. But with the arrival of Trikafta, many are facing a new, unexpected existential crisis. We explore the architecture of growing up sick, the trauma of living with an "expiration date," and the difficult reckoning that occurs when the horizon of your life suddenly shifts from years to decades.Featuring clinical psychologist Dr. Jodi Carrington, CF advocate Lauren Clift, and CF Canada CEO Kelly Grover, this episode pulls back the curtain on the psychological complexity of being "saved" and the urgent need to invest in the mind with the same intensity we've invested in the lungs.Main Topics & Key Moments1. The Architecture of Growing Up SickThe Parental Mask: Jeremie reflects on his parents' stoicism and the realization that their strength often involved "shattering" out of his sight."What Happened Here?" vs. "What's Wrong Here?": Dr. Jodi Carrington explains how reframing the conversation from pathology to experience creates the empathy necessary for healing.The Inside of Trauma: Why trauma isn't defined by the diagnosis itself, but by how the story unfolds within the family and community.2. Living with an Expiration DateThe Body Keeps Score: Lauren Clift shares her journey of middle-school anxiety attacks and the subconscious ways she absorbed the fragility of her life.Financial & Life Planning: Jeremie discusses the "freedom" of having no foundation—spending every dollar and avoiding long-term plans because tomorrow wasn't guaranteed.The Identity Crisis: What happens when the "honeymoon phase" of a new drug ends and you realize your entire identity was built around dying young?3. The Trikafta ReckoningSurvivor's Guilt: Addressing the "cruel math" where 90% of the community benefits from Trikafta while 10% are left waiting, and the heavy emotional weight carried by those who received the miracle.The Hospital Visit Realization: Jeremie recounts the moment a nurse suggested a psychologist and a friend's simple acknowledgment—"That must be really hard"—broke the emotional floodgates.4. What Do We Do Now?CF Canada's Mental Health Focus: Kelly Grover discusses Peer Connect, a pilot program designed to help patients, dads, and caregivers "say the unsayable" together.The Optimal Human Operating System: Dr. Carrington outlines the four pillars needed for post-traumatic growth: Attachment, Regulation, Community, and Role/Purpose.Key Quotes"When a miracle drug gives you back your future, you still have to figure out what to do with a mind that was built for a much shorter life." — Host"Trauma has nothing to do with what happened to you. It's all about what happens inside of you." — Dr. Jodi Carrington"The scary part isn't thinking about me dying. The scary part is that other people think I'm dying. I'm trying to process the feelings of... I could have died." — Lauren Clift"We have invested billions of dollars into the biology... but the mind? The mind is still catching up. If we don't invest in that frontier... we will lose people not to their lungs, but to their loneliness." — Host Hosted on Acast. See acast.com/privacy for more information.

Some people with cystic fibrosis have seen life-changing results from Trikafta, but not every patient responds to the treatment. The University of Kansas Health System looks at the research helping the group of CF patients who have been left behind by current therapies. Dr. Steve Stites is joined by cystic fibrosis patient Jason White, pulmonologist and critical care specialist Dr. Joel Mermis, and Dr. Matthias Salathe, Chief Research Officer for the University of Kansas and Chief Science Officer of The University of Kansas Health System. Together, they explain how CFTR mutations, theratyping, swab testing, and gene therapy clinical trials may help identify new treatment options for people living with cystic fibrosis and those with the common F508del mutation.

Episode Summary: The Impossible MilestonesThe Season 2 premiere of Breathless explores a biological and existential revolution. For decades, Cystic Fibrosis was defined by a "physical perimeter"—a list of things that were simply impossible. But with the advent of transformative drugs like Trikafta, those walls are vanishing.Host Jeremie opens with a visceral comparison between a 2017 spiritual "holotropic breathing" experience and the 2021 pharmaceutical miracle of his first dose of Trikafta. We then hear from elite athletes who shattered expectations long before the "miracle drug" existed, and we look at how Cystic Fibrosis Canada is pivoting its entire mission from "extending life" to supporting a life "without limits."Main Topics & Key Moments1. The Gateway to BreathingSalt Spring Island (2017): Jeremie describes a profound meditative state where, for a moment, the "heavy, wet coat" of CF was stripped away.The Blue Pill (2021): The "impossible milestone" found in a blister pack. Jeremie recounts the first morning he woke up without the "wet rattle" in his lungs, realizing his biology had been rewritten.2. The Science of the "Flow"The Broken Valve: Dr. Paul Eckford (Chief Scientific Officer, CF Canada) explains the CFTR protein.Chloride, Salt, and Water: A breakdown of why CF lungs create "concrete" mucus and how new modulators open the cellular gates to let water flow, clearing the airways.3. Shattering the Finish Line: Lisa BentleyThe Undiagnosed Athlete: Lisa won 11 Ironman championships despite having CF, including a period where she didn't even know she had the disease.The "Superpower": How meeting children with CF gave Lisa a purpose beyond winning, leading to her "lights out" fourth-place finish at the Hawaii Ironman World Championships while battling a chest infection.4. The Power of Delusion: Sophie Grace HolmesThe Two-Year Warning: At 19, Sophie was told she had two years to live. Her response? A bucket list that started with summiting Mount Kilimanjaro with 50% lung function.36 Marathons in 36 Days: Sophie's journey from a nationally ranked sprinter to an ultra-endurance icon, and her shock at how "easy" running became after Trikafta boosted her lung function to 110%.5. The Existential WhiplashFrom Pediatric to Geriatric: Kelly Grover (CEO, CF Canada) discusses the challenge of pivoting a non-profit toward supporting adult needs like housing, nutrition, and retirement.The Paradox of Progress: Why mental health challenges (anxiety and depression) are intensifying even as physical health improves. Jeremie describes the "existential whiplash" of having to plan for a future he never expected to have.Key Quotes"Trikafta didn't just open my airways; it dismantled the perimeter. The walls I spent thirty years banging against have simply… vanished." — Host"I am going to be the best sick person on that start line. And what I might lack in lung health, I'm going to make up for in heart." — Lisa Bentley"I didn't even realize I was struggling my whole life because I didn't have the opportunity to actually feel what it's like to actually breathe properly." — Sophie Grace Holmes"I might have a bit of a freakout if all of a sudden the world was my oyster with no restrictions on it." — Kelly Grover Hosted on Acast. See acast.com/privacy for more information.

Very young children with cystic fibrosis could soon access funded medicines with the potential to add decades to their lives. Christchurch couple Edward and Emily Harrington-Ashton's 20 month-old- son, Noah, has cystic fibrosis. They say the drug will be life changing for their son. Edward spoke to Lauren Crimp.

Families of young children with cystic fibrosis are celebrating a Pharmac proposal to widen access to the drug Trikafta, which is out for consultation. It's been funded for children over 6 since 2023, but the agency's now proposing extending it to under-sixes. Liam Rooney's child has cystic fibrosis and he says his family was looking for other ways to get it. "I'd already spoken to my boss over in Australia about the potential for us to move over if things got worse." LISTEN ABOVESee omnystudio.com/listener for privacy information.

Tonight on The Huddle, journalist Clare de Lore and broadcaster Mark Sainsbury joined in on a discussion about the following issues of the day -and more! Families of young children with cystic fibrosis are celebrating a Pharmac proposal to widen access to the drug Trikafta, which is out for consultation. How good is this? Donald Trump has laid the groundwork or a future deal involving Greenland and dropped proposed tariffs on NATO countries. What do we make of this? Do we see Trump getting his way here? A new Taxpayers' Union-Curia poll dropped - and it indicates the current coalition Government could get re-elected. NZ First took a leap to 11.9 percent, what can we take from this? LISTEN ABOVESee omnystudio.com/listener for privacy information.

What does it mean to live with cystic fibrosis (CF) in Amish and Mennonite communities, where many families don't use phones, computers, or even electricity? For some, this means relying on handwritten letters for communication, trying herbal remedies before conventional medicine, and declining treatments like lung transplants or in vitro fertilization because of cultural and religious beliefs.Update State CF Center (Syracuse, NY) Social Worker Lejla Bush, who has worked with the CF community for over a decade, shares how Amish and Mennonite families navigate CF while staying true to their traditions. She explains the unique challenges, from financial hurdles without health insurance, to hospital care that must adapt to cultural practices, and the vital role of community support in helping families face this disease.Most importantly, Lejla reminds us that while the cultural context is different, Amish and Mennonite parents hold the same hopes, fears, and love for their children as any other family affected by CF.This episode opens a window into the powerful intersection of culture, medicine, and resilience.We did a Q and A with some people in the Amish and Mennonite communities. Thanks to Lejla for sharing so much information about the Amish and Mennonite communities.To see a letter of Q and A with one of the Amish patients click here: https://thebonnellfoundation.org/wp-content/uploads/2025/09/AmishLetter.jpg Please like, subscribe, and comment on our podcasts!Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website:https://thebonnellfoundation.orgEmail us at: thebonnellfoundation@gmail.com Watch our podcasts on YouTube: https://www.youtube.com/@laurabonnell1136/featuredThanks to our sponsors:Vertex: https://www.vrtx.comViatris: https://www.viatris.com/en

Mark Fadel, MD joins this episode of Head & Neck Innovations to discuss recent advances and innovations he is excited about as he starts his practice in pediatric otolaryngology. Advancements in medical treatments like Dupixent and Trikafta are transforming care for children with allergic rhinitis and cystic fibrosis—reducing the need for surgery and improving outcomes.

Jennifer McKinnon is fierce. Doctors said Jennifer wouldn't live past the age of three.Today, she's a single mom of twins, an unstoppable advocate who's raised over a million dollars for cystic fibrosis research, and the founder of Just One More Breath. Her story is proof that while CF is tough, Jennifer is tougher, and that hope, when held fiercely, can change everything.Jennifer was born in the 1970s with cystic fibrosis, a time when the disease was still cloaked in uncertainty and fear. Few children with CF lived to see adulthood, and the treatments that exist today were still decades away. From the very beginning, every breath she took was a small act of defiance. Hospital rooms became her playgrounds, IV poles her silent companions, and the steady rhythm of nebulizers the soundtrack of her childhood.But even in those sterile rooms filled with machines and medicine, Jennifer's spirit burned bright. She refused to let CF define her — or defeat her. Through endless hospital stays, setbacks, and the exhausting daily grind of treatments, she found reasons to laugh, to dream, and to keep fighting. Every milestone, every birthday, every deep breath, became a victory.Then came 2019, a year that would rewrite her story once again. With the arrival of Trikafta, the groundbreaking CF modulator, Jennifer's world shifted. Suddenly, life felt lighter — her lungs stronger, her body more capable. For the first time, she could picture a future not limited by her disease but expanded by possibility. She could plan, imagine, and live, truly live, in ways she once only dreamed of.Yet Jennifer's story isn't just about survival; it's about transformation. Rather than turning inward, she turned outward — channeling her strength into action. She's raised over a million dollars for cystic fibrosis research, driven by the belief that every dollar brings us closer to a cure. She became a mother to twins through the extraordinary gift of her sister, proving that family and love can find a way even when the odds seem impossible.Today, Jennifer stands as a single mom, a tireless advocate, and the voice behind Just One More Breath, a platform dedicated to raising awareness, sharing stories, and inspiring others to keep pushing forward ... one breath at a time.Her journey is a powerful reminder that life with cystic fibrosis is unpredictable, yes, but it is also breathtakingly beautiful. It's a life lived with intention, courage, and grace. Jennifer's story teaches us that even when the odds seem insurmountable, resilience can turn pain into purpose, and hope can carry you through the darkest storms.Because for Jennifer, every breath is more than survival.It's a celebration of strength, of love, and of the limitless power of the human spirit.Jennifer on IG: https://www.instagram.com/just.onemorebreath/?hl=enWebsite: https://jennifer-mckinnon.com/?  Please like, subscribe, and comment on our podcasts!Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website:https://thebonnellfoundation.orgEmail us at: thebonnellfoundation@gmail.com Watch our podcasts on YouTube: https://www.youtube.com/@laurabonnell1136/featuredThanks to our sponsors:Vertex: https://www.vrtx.comViatris: https://www.viatris.com/en

The parents of an Auckland toddler say they're living in hope their wee boy will soon have access to a "miracle" life-extending treatment for his cystic fibrosis. Pharmac currently funds the drug Trikafta for those six years and older - but it's now considering including two to five-year-olds. Lauren Crimp reports.

What's it like getting pepper-sprayed when you have a chronic lung disease? A homicide detective knows what's up. This week, we sit down with a guest who defies expectations: Jeremy, a homicide detective living with Cystic Fibrosis (CF). We dive into the unique challenges and surprising advantages of managing a demanding, high-stress career alongside a chronic illness. He opens up about the progression of his CF, the profound impact of the life-changing drug Trikafta, and his perspective on life expectancy before and after the medication. You can watch this entire episode over on YouTube.Follow Sickboy on Instagram, TikTok and Discord!

What's it like getting pepper-sprayed when you have a chronic lung disease? A homicide detective knows what's up. This week, we sit down with a guest who defies expectations: Jeremy, a homicide detective living with Cystic Fibrosis (CF). We dive into the unique challenges and surprising advantages of managing a demanding, high-stress career alongside a chronic illness. He opens up about the progression of his CF, the profound impact of the life-changing drug Trikafta, and his perspective on life expectancy before and after the medication. You can watch this entire episode over on YouTube.Follow Sickboy on Instagram, TikTok and Discord!

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into some of the most significant shifts and strategies shaping our industry.Novartis's acquisition of Avidity Biosciences for a staggering $12 billion marks a pivotal moment in the pharmaceutical landscape this year. With this acquisition, Novartis underscores its commitment to bolstering its neuromuscular disease pipeline. Avidity Biosciences has made a name for itself with its cutting-edge RNA therapeutic technologies, particularly its Antibody Oligonucleotide Conjugates (AOCs). This platform uniquely combines monoclonal antibodies with oligonucleotides, enhancing precision in targeting specific cell types. The integration of Avidity's technology into Novartis's research efforts could accelerate the development of new therapies, potentially transforming patient care with more effective and targeted treatment options. This move not only highlights the industry's focus on specialized therapeutic areas but also anticipates future advances in RNA therapeutics, extending beyond neuromuscular disorders to areas like oncology.In a similar vein, the FDA has shown its willingness to reconsider drugs that previously faced setbacks. GSK's Blenrep has made a return to the U.S. market after receiving approval for treating certain myeloma patients. This approval is particularly noteworthy given the drug's earlier negative advisory committee vote and postponed decision. It marks a significant rebound for GSK's oncology portfolio and reflects the FDA's dynamic approach towards drugs that show potential in specific therapeutic combinations.Meanwhile, Sanofi continues to make waves with Dupixent, achieving over €4 billion in quarterly sales due to its expanded indications. This success contrasts with a decline in Sanofi's vaccine sales, demonstrating shifting dynamics within pharmaceutical portfolios where biologics and specialty drugs are increasingly pivotal. Sanofi's recent financial report highlighted a notable 17% drop in vaccine sales due to reduced demand and pricing challenges in Europe. In response, companies must navigate fluctuating public health demands and economic pressures effectively.On the global stage, efforts to make transformative therapies like Vertex's Trikafta more accessible are gaining momentum through innovative trade-policy workarounds. A buyers club aims to introduce a lower-cost alternative produced by Bangladesh's Beximco, highlighting ongoing challenges and creative strategies in global drug accessibility.Roche's expansion through Chugai's $200 million M&A deal for an IgA nephropathy asset underscores the strategic importance of regional markets in driving growth. Similarly, Lonza's acquisition of a California biologics site aligns with its goals to meet increasing biomanufacturing demands.The industry is also adapting to technological advancements, with AI integration into life sciences commercialization being touted as a frontier for growth. Despite this potential, many organizations remain unprepared to harness AI fully. Leading companies embedding AI solutions aim for measurable outcomes that could significantly drive strategic decision-making and operational efficiencies.Eli Lilly's acquisition of Adverum Biotechnologies aligns with its strategic interests in gene therapy, focusing on promising therapeutic programs that address unmet medical needs. This acquisition centers around Ixo-vec for wet age-related macular degeneration (AMD), highlighting broader industry trends towards investing heavily in innovative therapies that address unmet needs.Conversely, Sanofi's halt on an RSV vaccine development highlights the inherent risks in vaccine development pipelines. Meanwhile, Regeneron's decision to discontinue a CAR T candidate acquired from 2seventy bio showcases ongoing reassessment witSupport the show

Pulmonologist Michael J. Welsh, MD, is the co-recipient of the 2025 Lasker-DeBakey Clinical Medical Research Award for his research on cystic fibrosis. In this interview, he discusses his early clinical encounters with the disease, the foundational work that led to the lifesaving drug Trikafta, and future directions for cystic fibrosis treatment. Related Content: How Cystic Fibrosis Went From Fatal to Treatable Rewriting the Chapter on Cystic Fibrosis

欢迎收听雪球出品的财经有深度，雪球，国内领先的集投资交流交易一体的综合财富管理平台，聪明的投资者都在这里。今天分享的内容叫聊聊生物医药这个行业，来自LTLyra。生物医药这个行业，两类公司，biotech，biopharma。这两类公司之间，隔着天堑，或者说，这两类公司，需要的能力，是截然不同的。生物医药这个行业，基本可以分成三段能力：研发，临床和商业化。所谓biotech，基本就是只具备研发，强一点的，加上部分或者全部临床，顶天了。所谓biopharma，就是具备以上三段能力的完整体。研发能力，放在全球都是一样的。但临床和商业化能力，国内和海外，也是隔着天堑。实际上，临床和商业化能力，很大程度上，是关联的。一个药，做临床的医生和医院，基本和成药以后销售药品的医生和医院，是一波，当然，临床成功，需要把药卖到更多的医院去。一个只有研发能力的biotech，站在一个在这个市场商业化了很多产品的pharma之前，基本就跟一个小孩站在一个大人跟前一样。不要小看临床和商业化的资源，这是这个行业最核心的资源，比研发能力还重要。研发能力，可以用钱买到；临床和商业化资源，本质上，是很多年，开发了很多药，缓慢跟医生和医院建立的信任关系。所以，全世界，这个行业的商业模式就是，biotech负责研发和早期临床；如果数据优异，把产品 B D 给pharma，由后者负责后期临床和商业化，这是这个行业最有效率的运行方式。生物医药这个行业，在研发端，天然是逆集中的。你永远不知道，下一个最好的药，在哪个犄角旮旯的实验室里。所以，我们就看到，手握商业化资源的跨国公司，拿着美元，全世界买买买。Biotech，实现股东价值的方式，只有三种。第一，把产品卖给pharma；第二，把自己卖给pharma；第三，自己成为pharma。以上三种，难度递增，第三种更是地狱级难度。Biopharm，实现股东价值的方式，只有一种，就是卖药。不管这个药是自己研发的，还是别人研发出来，然后自己买来的。那么，如何从biotech晋级成biopharma呢？大体上，只有一种途径，就是靠一款及其优秀的大药打天下，逐步站稳脚跟，靠这个大药提供源源不断的现金流，支撑不断的研发，如果后续管线能够跟上，逐步成为biopharma。这个说起来容易，做起来极难。因为这行业竞争非常激烈，biotech开发出一款好的产品，很难有足够的时间靠自己推动商业化。同一个产品，pharma从三期临床推到商业化，可能2年时间，但biotech，2年时间，三期临床，入组可能还没有完成。除非这个药好到没有竞品，可以自己慢慢搞，否则，这行业，慢就是死路一条。同靶点同机制的药，如果疗效没有代差，第一个上市的吃肉，第二个上市的喝汤，后面的，基本以收回研发成本为目标。举几个历史上成功越过这道天堑的例子。福泰制药。依靠罕见病药品，全球首款针对具有特定基因突变人群的囊性纤维化根本原因进行治疗的药物KALYDECO站稳脚跟，然后，在这个领域，持续迭代出ORKAMBI和TRIKAFTA，才站稳biopharma的地位。TRIKAFTA，2024年已经是百亿美金销售的大药。就是靠这个强大的现金流，福泰制药才发育起来。根本上，福泰制药能发育起来的原因，是他从罕见病切入，没有对手，才找到发育的时间。但是，福泰制药1989年成立，到2012年KALYDECO上市，中间吃土了20多年……再生元。依靠治疗湿性黄斑变性等眼科疾病的Eylea打天下。Eylea，百亿美元年销售额的大药。依靠Eylea的现金流，开发出大名鼎鼎的度普利尤单抗，又一个百亿美金峰值的大药。但是，再生元1988年成立，Eylea 2011年上市，中间也是20多年……一个“国产”样本，百济神州。同样，一款大药，泽布替尼打天下。在礼来的变构 B T K 抑制剂三期临床折戟，阿斯利康阿卡替尼联合维奈克拉三期临床数据差强人意的背景下，没人怀疑泽布替尼是50亿销售峰值的大药了吧？百济神州这个样本有点特殊，他能在这么短的时间，从biotech晋级成biopharma，除了泽布替尼这款大药，另外一个依靠的就是中美港三地资本市场。几年时间，百济在三个市场融资百亿美金，用这些钱，疯狂的撕开欧美市场的一个口子，把泽布替尼推了进去。这个事情，前无古人，后面也不会有来者。再者，这个世界上，即使强如Genmab，开发出百亿美元销售峰值的达雷木单抗，和几十亿美元销售峰值的奥托珠单抗。也只能无奈把两个产品分别BD给强生和诺华，自己依然是一个biotech。生物医药这个行业，国内和国外，就是两个市场。同一个药，美国药价是国内的十倍到几十倍；美国药价是欧洲的几倍；事实上，这个产业，就是美国市场补贴了全世界的生物医药研发。所以，判断一家生物医药公司的上限，有且仅有一个标准，就是能不能把药卖到美国去。国内药企，最强的那批，就是能把一个或者几个大药，卖到美国去的公司。做一个好药挺难，但想把这个药卖到美国去，更难。尤其，在当前这种地缘政治环境下。但是，这个行业，天然就有一种规避这种地缘政治风险的模式，叫做授权合作。通过授权，让渡部分权益，变现自己的研发能力。前面为啥说百济神州前无古人，后无来者？原因就是，他在一个最好的时机，即中美关系尚可，公开互怼前夕，融到了很多钱，完成了从biotech到biopharma的惊险一跃。况且，可能大多数人不知道的是，百济神州，其实是一家披着中国外衣的美国公司。他家老板，是纯正的出生在美国的白种人；他家股东，大多都是欧美资金；百济，其实就是借中国优秀的研发能力，走出来的一家美国公司。这么多年，真正从国内走出去，在全球成为biopharma的，其实只有一家半公司，如果百济神州算一家的话。另外的半家，是金斯瑞孵化出来的传奇生物。传奇生物，成为biopharma，靠的也是一款大药，预期百亿美金销售峰值的西达基奥伦塞，但传奇生物只有这个药一半的权益。每次说到传奇生物，经常有人认为金斯瑞拆分传奇生物美股上市，通过利益输送，传奇生物股权越来越低。但其实，开发西达基奥伦塞这个百亿美金销售峰值的大药，前前后后，传奇生物投入了很多资金。不通过分拆传奇生物美股上市，金斯瑞2、300亿港币市值的体量，在羸弱的港股，很难能融到足够的钱。西达基奥伦塞这个药，传奇生物和强生一边一半权益。包括临床费用，产能建设，这些钱，都要一边一半。为开发这款药，到目前为止，双方已经累计投入30多亿美金。一边16亿美金以上。传奇生物，除了拿到强生给的首付款3.5亿美金，还通过美股IPO以及增发，融资十几亿美金，刚刚够这个产品的开发投入。事实上，金斯瑞当时做出了最合乎逻辑的选择，就是分拆传奇生物，让他独立融资发展。如果当时他抱着这个金蛋不松手，因为缺钱跟强生搞出矛盾，耽误了这个药的开发，才是对全体股东利益的伤害。跟着强生开发一个百亿美金大药，在这个过程，传奇生物获得的最大隐形彩蛋，就是建立起了在海外的临床和商业化能力。这个能力，如果用金钱衡量，我想，可能是数十亿美金级别的。为啥强生出资150亿美金收购传奇生物，金斯瑞不卖？其实，150亿美金，是对西达基奥伦塞一半权益的合理报价。但传奇生物来说，他不仅只有这一个药，他还拥有难得的，作为一个全球biopharma的临床和商业化能力。这意味着，后面的管线，他都是有能力在海外独立商业化的，这个价值是无法衡量的。所以，说了这么多，知道怎么投生物医药公司了吗？不是非得能自主海外商业化大药的公司才能投。但是，要买那些市值和能力匹配，而且头脑清醒，战略正确的公司。如果一个几十亿港币市值的小公司，手里拿着几个还在临床前期的产品，然后每天畅想自己去海外卖药，那他的潜力是令人担忧的。但如果一家几十亿港币市值的小公司，手里有个已经临床三期，确定FIC或者BIC，且能有10亿美金级别销售峰值的产品，而且战略很清晰，就是要跟跨国公司合作开发这个产品，那就是极好的投资机会。但是，最好的那个投资机会，一定是那种手里握着大药，最好有百亿美金销售峰值的大药，而且，能正确衡量自己能力，战略选择正确的公司。比如，靠国内市场站稳脚跟，有稳定的基本盘，然后，通过合作走出国门，逐步培养自己的能力，依靠优秀的管线排布，慢慢往全球biopharma进化的公司。对于投资人来说，我们脑子一定要清醒，明白自己买的到底是什么。如果我们买的是就是一个biotech，但他搞不清自己的边界，偏偏想自己商业化产品，那就是个悲剧。这个行业，天然就不好懂，所以，搞点似是而非的概念，在牛市的掩护下，疯狂割韭菜的公司，需要我们谨慎辨别并作出理智的决断。

If Fierce Pharma Marketing’s annual list of the top 10 biggest potential drug launches of the coming year is any indication, biopharma may soon be in for a blockbuster boom. All together, the 10 meds that made the 2025 list stand to generate a whopping $29 billion in annual sales by the end of the decade. In this week’s episode of The Top Line, we dig into the report’s predictions. Fierce’s Andrea Park and Eric Sagonowsky take a deep dive into the top three drugs on the list—all of which had already snagged their first FDA approvals by this episode’s release—and highlight some of the prevailing trends from past years’ reports, including repeat entries, popular indications and drugs that never had the chance to meet their predicted potential. To learn more about the topics in this episode: Top 10 most anticipated drug launches of 2025 Vertex snags FDA nod for once-daily cystic fibrosis triplet Alyftrek as switch from Trikafta kicks off Datroway, 2nd ADC from AstraZeneca-Daiichi collab, wins first FDA nod in breast cancer Vertex scores FDA nod for long-awaited non-opioid pain reliever Journavx This episode is brought to you by Cencora. Learn more at cencora.com/breakthrough.See omnystudio.com/listener for privacy information.

In this episode of It Happened To Me, co-hosts Beth Glassman and Cathy Gildenhorn sit down with Laura Bonnell, a seasoned journalist with 25 years of experience in Detroit, founded The Bonnell Foundation in 2010 after her daughters, Molly and Emily, were born with cystic fibrosis (CF). Despite having ten siblings between them, Laura and her husband, Joe, had no idea they were carriers of the disease, as no one else in either family had been diagnosed with CF. Driven by her personal experience and professional expertise, Laura used her platform as a reporter to raise awareness about CF. Before establishing The Bonnell Foundation, she actively raised funds for the Cystic Fibrosis Foundation, participating in the Great Strides Walk fundraisers and speaking at CFF events. Bonnell also organized events like the first Celebrity Softball game with the Detroit Tigers' wives and launching the "Portraits of Cystic Fibrosis" calendars in 2003—both of which are integral to The Bonnell Foundation's fundraising efforts today.  Through her foundation, Laura continues to advocate tirelessly for those affected by CF, leveraging her journalistic skills to amplify the cause and support the CF community. Topics Covered: Understanding Cystic Fibrosis: What it is, how it affects the body, and the daily medical routines required to manage it. Personal Journey: Laura's experience with her daughters' diagnoses and the challenges her family faced. Advocacy Through Journalism: How Laura's skills as a journalist influenced her podcast, Living With Cystic Fibrosis, and her advocacy work. The Bonnell Foundation: From organizing the first Celebrity Softball game with the Detroit Tigers' wives to producing the "Portraits of Cystic Fibrosis" calendars, Laura discusses her foundation's mission and impact. Challenges and Progress: The evolving landscape of CF treatment, including the life-changing potential of medications like Trikafta. Community Support: How The Bonnell Foundation helps families navigate life with CF and fosters a sense of connection. Resources Mentioned: The Bonnell Foundation The Bonnell Foundation Facebook The Bonnell Foundation X The Bonnell Foundation Instagram Living With Cystic Fibrosis Podcast Connect with Us:  Stay tuned for the next new episode of “It Happened To Me”! In the meantime, you can listen to our previous episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “It Happened To Me”.    “It Happened To Me” is created and hosted by Cathy Gildenhorn and Beth Glassman. DNA Today's Kira Dineen is our executive producer and marketing lead. Amanda Andreoli is our associate producer. Ashlyn Enokian is our graphic designer.   See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, ItHappenedToMePod.com. Questions/inquiries can be sent to ItHappenedToMePod@gmail.com.   

Das Medikament Trikafta hat das Leben unzähliger Menschen zum Besseren gewendet, die an der Erbkrankheit Cystische Fibrose (CF) leiden. Doch das Märchen hat eine Kehrseite: Die Behandlung ist enorm teuer. Sprengt ein solches Medikament das solidarische Gesundheitssystem? Rund 200'000 Franken kostet es offiziell pro Jahr, eine CF-Patientin bzw. einen CF-Patienten mit Trikafta zu behandeln. Zumindest ist dies der offizielle Preis. Es sind wiederkehrende Kosten, die das Schweizer Gesundheitssystem gemäss einer Kosten-Nutzen-Berechnung mit 160 Millionen Franken belasten. Ist es fair, so viel Geld für relativ wenige Betroffene auszugeben? Stimmt der offizielle Preis von Trikafta überhaupt? Welchen Hebel haben Gesundheitsbehörden wie das BAG, um sich gegenüber den Herstellern – im Fall von Trikafta die US-Firma Vertex – bei Preisverhandlungen zu behaupten? Mit BAG-Vizedirektor Thomas Christen und der Medizinethikerin Tanja Krones (Universität Zürich) spricht Irène Dietschi.

Das Medikament Trikafta hat das Leben unzähliger Menschen zum Besseren gewendet, die an der Erbkrankheit Cystische Fibrose leiden, unter ihnen die Schwestern Maurane (27) und Sarah (29). Doch nicht allen Betroffenen hilft Trikafta. Und: Das Medikament ist viel zu teuer. Die Kindheit von Maurane und Sarah aus dem solothurnischen Hägendorf ist ein permanenter Ausnahmezustand – Spitalaufenthalte, tägliches Inhalieren, Physiotherapie. Doch dann katapultiert das Medikament Trikafta die Schwestern in ein fast normales Leben. Das Glück hat seinen Preis: Maurane und Sarah sind traurig für jene, denen das neue Medikament nicht hilft. Und: Die Behandlung ist enorm teuer. Die Vergütung von Trikafta durch die Krankenkassen ist auf den 31. Januar 2025 befristet. Wie geht es weiter? Im Podcast zu hören sind: · Sarah Dincer und Maurane Wüthrich, Betroffene der Erbkrankheit Cystische Fibrose · Simon Wieser, Professor und Leiter des Instituts für Gesundheitsökonomie der Zürcher Hochschule für Angewandte Wissenschaften ZHAW · Thomas Christen, Vizedirektor Bundesamt für Gesundheit BAG Bei Fragen, Anregungen oder Themenvorschlägen schreibt uns: kontext@srf.ch Autorin: Irène Dietschi Host: Bernard Senn Produktion: Anna Jungen Technik: Serge Krebs

You've probably never heard of Trikafta — unless you or someone you love lives with cystic fibrosis. The drug has fundamentally changed cystic fibrosis care, extending life expectancy by decades. But the drug costs $326,000 a year — and requires people to be on it for life. Abdul reflects on the central challenge at promoting life-saving innovations that the people who need them can afford. Then he interviews Dr. Michael Boyle, President and CEO of the Cystic Fibrosis Foundation about the science behind Trikafta and the work to make it universally affordable. This show would not be possible without the generous support of our sponsors. America Dissected invites you to check them out. This episode was brought to you by: Marguerite Casey Foundation: Sign up for their new Summer School program at CaseyGrants.org/SummerSchool. Rhone: Upgrade your closet with Rhone and use AD to save 20% at https://www.rhone.com/AD.

They say the only constant in life is change, but what happens when the change you're given feels like a double-edged sword? In this episode, the boys sit down with Connie, a young woman whose life with Cystic Fibrosis has taken a sharp turn thanks to Trikafta. She shares the raw realities of navigating a new chapter where her body, mind, and future are all in flux. From the ripple effects of this life-altering drug to the unexpected burden of added years, Connie explores the challenges of redefining herself and seeking community in a world that suddenly feels both more hopeful and isolating.Catch the full video version of this episode on YouTube!Follow Sickboy on Instagram, TikTok and Discord

They say the only constant in life is change, but what happens when the change you're given feels like a double-edged sword? In this episode, the boys sit down with Connie, a young woman whose life with Cystic Fibrosis has taken a sharp turn thanks to Trikafta. She shares the raw realities of navigating a new chapter where her body, mind, and future are all in flux. From the ripple effects of this life-altering drug to the unexpected burden of added years, Connie explores the challenges of redefining herself and seeking community in a world that suddenly feels both more hopeful and isolating.Catch the full video version of this episode on YouTube!Follow Sickboy on Instagram, TikTok and Discord

Cambrey Vasconez White is the mother of toddler Rowland, who has Cystic Fibrosis. If you listened to our podcast with Vicky Maldonado, they have similar  struggles.  Cambrey is also working to find an equitable approach to rare mutations in the U.S. and Canada.  These two women connected, as you'll hear, because their sons share the same mutation.Rowland received  access to a CF modulator after a battle with her insurance company.  Her son's mutation is not on the FDA approved list for Trikafta…. even though there is proof that Trikafta will work on her son's mutation.To follow Cambrey go to @cfadvocacyYou can email her: cambreywhite@gmail.com Please like, subscribe, and comment on our shows, wherever you get your podcasts.Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website:https://thebonnellfoundation.orgEmail us at: thebonnellfoundation@gmail.com Thanks to our sponsors:Vertex: https://www.vrtx.comViatris: https://www.viatris.com/en

In our final episode, we explore Dorothy Andersen's legacy — what she left behind and how her work has lived on since her death. Describing her mentor's influence on her life and career, Dr. Celia Ores gives us a rare look at what Dr. Andersen was really like. We then turn to researchers, physicians, and patients, who fill us in on the many areas of progress that have grown out of Dr. Andersen's work. These major developments include the discovery of the cystic fibrosis gene, the tremendous impact of the drug Trikafta, and the lifesaving potential of gene editing techniques. We end the episode with an update on the effect Trikafta has had on the lives of many CF patients, who can now expect to live a normal life. 

When science fiction becomes a medical reality, the journey is anything but simple. Kelly Grover, CEO of Cystic Fibrosis Canada, dives into how Trikafta, the breakthrough CF drug, is revolutionizing treatment by targeting its root cause. While Trikafta brings significant physical health benefits, Kelly and Jeremie discuss the emotional and mental health challenges that arise as patients navigate new possibilities and uncertainties. The discussion highlights CF Canada's relentless efforts to support the CF community, advocating for research, improving access to treatments, and addressing the unique needs of those with rare mutations. Stick around for the end of the episode as Jeremie introduces the fellas to the work of the late/great Bob Flanagan - a performance artist who lived with CF and had a wild portfolio of art blending CF with BDSM.For more info on Cystic Fibrosis visit here.Catch the full video version of this episode on YouTube!Follow Sickboy on Instagram, TikTok and Discord.

When science fiction becomes a medical reality, the journey is anything but simple. Kelly Grover, CEO of Cystic Fibrosis Canada, dives into how Trikafta, the breakthrough CF drug, is revolutionizing treatment by targeting its root cause. While Trikafta brings significant physical health benefits, Kelly and Jeremie discuss the emotional and mental health challenges that arise as patients navigate new possibilities and uncertainties. The discussion highlights CF Canada's relentless efforts to support the CF community, advocating for research, improving access to treatments, and addressing the unique needs of those with rare mutations. Stick around for the end of the episode as Jeremie introduces the fellas to the work of the late/great Bob Flanagan - a performance artist who lived with CF and had a wild portfolio of art blending CF with BDSM.For more info on Cystic Fibrosis visit here.Catch the full video version of this episode on YouTube!Follow Sickboy on Instagram, TikTok and Discord.

Very cool episode this week. Today I sit down with a true one of one in the podcasting world, Jeremie Saunders from CBC's Sickboy podcast, to explore his experiences as a man living with Cystic Fibrosis (CF), the most common fatal genetic disease affecting Canadian children and young adults. Jeremie shares how he learned of his diagnosis as a child and how it shaped his views on life after learning that he'd be unlikely to see his 35th birthday. He explores the nuances of living life faced with his mortality through the lens of mental health and provides an authentic look into the highs and lows associated with his disease and the subsequent release of a life-saving drug in Trikafta.Check out SickBoy on Apple PodcastFollow Jeremie on InstagramCheck out CF Canada's Podcast BreathlessFollow Life's a Wreck on InstagramFollow Kyle on InstagramPlease explore local mental health resources. You are worthy of the health that you seek. If you are in crisis please dial 9-8-8 for a national, free, 24/7 Suicide Crisis Line. Hosted on Acast. See acast.com/privacy for more information.

The drug Trikafta has changed the lives of many Canadians living with cystic fibrosis. But in many other parts of the world, the drug is either not available — or if it is, it costs hundreds of thousands of dollars a year. That's why Cheri Nel, who is based in Johannesburg, is suing American pharmaceutical company Vertex over its patent and price tag.

In this episode, Amy Sylvis sits down with Gunnar Esiason, a prominent figure in the cystic fibrosis (CF) community. Gunnar delves into his remarkable journey, recounting his diagnosis at a young age and his family's commitment to raising awareness and funds for CF research through the Boomer Esiason Foundation. Despite facing challenges, Gunnar's passion for patient empowerment and early-stage drug development led him to pivotal roles in healthcare and advocacy.  Despite facing challenges with CF, Gunnar's resilience and determination led him to pursue higher education, ultimately earning an MBA and MPH. Throughout the conversation, Gunnar reflects on his life before and after receiving the breakthrough drug TRIKAFTA, describing what it was like taking his first dose. He shares personal insights into balancing work, family, and supporting the CF community, acknowledging the interconnectedness of these aspects in shaping his abundant life.  His story serves as an inspiration for all – showcasing the power of perseverance and advocacy in the face of adversity. More about Gunnar: Gunnar Esiason is a cystic fibrosis and rare disease patient leader, who is passionate about early-stage drug development, patient empowerment and health policy. He is the head of patient engagement at RA Capital's venture group, RA Ventures. Professionally, he developed a patient engagement platform for a medical nutrition company, built a venture philanthropy practice at the Boomer Esiason Foundation which has yielded more than $160 million raised for the fight against cystic fibrosis since he was diagnosed with the disease in 1993. He has consulted on clinical trial development, a real-world evidence population health study, and a cystic fibrosis-specific mental health and wellness screening tool. His blog has amassed nearly 1 million page views since 2015. His podcast, the State of Health, is available on all streaming platforms. Gunnar holds an M.B.A. from the Tuck School of Business at Dartmouth, where he was a Wilson Scholar and received the Julia Stell Award, an M.P.H. from the Dartmouth Institute for Health Policy & Clinical Practice, and a B.A. from Boston College. His health policy opinions have been featured in the Wall Street Journal, USA Today, The Hill, and STAT News. Connect with Gunner: esiason.org twitter.com/g17esiason Connect with Amy Sylvis: sylviscapital.com sylviscapital.com/webinar  linkedin.com/in/amysylvis

Canadian, Vicky Maldonado is the mother of a young boy living with a rare genetic form of CF. Sebastian is 6 years old, and a twin. The road to diagnosis was challenging. Sebastian was diagnosed with two rare CF genes after repeated respiratory infections and two hospitalizations. “We were told that Sebastian likely didn't have CF because he's “not White”— his parents are Canadian-born Latinos from Ecuador. Though CF affects all races and ethnicities, there's still a common misconception among health-care providers that it is a predominantly White disease.”Vicky and her husband were devastated to learn that their son would not have access to the life-changing and life-saving drug Trikafta because he has two rare mutations. Due to small populations in rare mutations, clinical trials are often not feasible. Other countries are using in vitro data to provide access to those with rare and ultra-rare mutations while approximately 200 Canadians with CF are being left behind.This is not a new problem in Canada, the pathway to access to new and innovative therapies is fraught with challenges. A broader regulatory approach is needed to support implementation of the National Strategy for Drugs for Rare Diseases, and cystic fibrosis is an example of this need. There are hundreds of disease-causing mutations, some with only a handful of patients worldwide. Health Canada can improve access to rare disease medications like Trikafta by using patient and laboratory in vitro data and by developing a regulatory model that permits bulk approvals of gene mutations that can respond to precision medicines like Trikafta. Please like, subscribe, and comment on our shows, wherever you get your podcasts.Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website:https://thebonnellfoundation.orgEmail us at: thebonnellfoundation@gmail.com Thanks to our sponsors:Vertex: https://www.vrtx.comViatris: https://www.viatris.com/en

Joining us this week is long-time friend of Gavin's, Nick Laing, to talk about his struggles with cystic fibrosis, his upcoming clothing line, and working on music with Ukdian (Gavin's band) follow Nick on Instagram @withinanickoftrikafta

Bernie Martin is a Writer, Creative Consultant and, most importantly, Mother of a CF Fighter. After 15 years working as a Copywriter and Creative Director in some of Ireland's top advertising agencies, she started her own consultancy called The Salty Pen in 2018. This move was born out of a desire to have more flexibility around caring for her little lady with CF, who she describes as her muse, her strength, her drive, her everything! Bernie has written about the challenges facing CF families on her blog My Little Miss Salty, and she has written for The M Word and MummyPages. She has worked on a voluntary basis as a CF patient advocate in CHI Temple Street and as a campaigner during the #YesOrkambi campaign in 2016/17, with the support of Rothco, the advertising agency she worked with at the time. She has been a speaker at the Cystic Fibrosis Ireland Conference and at the new parent information day in Temple Street, as well as featuring in Humans of Dublin by Peter Varga.Bernie and her daughter, Eva, recently collaborated with the University of Notre Dame in the 100th episode of their long running series, ‘What would you fight for?' In this case, the fight is for new Cystic Fibrosis treatments. Bernie and her husband Dave live in Dublin, Ireland.Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website: https://thebonnellfoundation.orgBonnell Foundation email: thebonnellfoundation@gmail.comThanks to our sponsors:Vertex:  https://www.vrtx.comGenentech: https://www.gene.comViatris: https://www.viatris.com/en

This week the boys are delving deep into the jaw-dropping findings of a recent study on near-death experiences, where the line between life and the great beyond gets a little blurry. A recent study shows how patients displayed brain activity in the delta, theta, alpha, and beta ranges, indicating possible conscious activity over 60 minutes AFTER their heart stopped! Maybe ghosts are real? Brian showed up with a Gen Z haircut but he's behind in the race for the most trendy 'do. One rapper from Mexico had hooks implanted into his head so he could rock a full head of bling… and to be quite honest, it looks pretty sick. Trikafta won a big ol' prize for extending Jer's life! Thai food is delicious but one dish may kill you with one bite and for WHAT THE HELLTH!?! The boys cover an insanely shocking story of one woman who's blood was so toxic that it landed multiple healthcare providers in the hospital after trying to save her life! Join the post-episode conversation over on Discord! https://discord.gg/expeUDN

This week the boys are delving deep into the jaw-dropping findings of a recent study on near-death experiences, where the line between life and the great beyond gets a little blurry. A recent study shows how patients displayed brain activity in the delta, theta, alpha, and beta ranges, indicating possible conscious activity over 60 minutes AFTER their heart stopped! Maybe ghosts are real? Brian showed up with a Gen Z haircut but he's behind in the race for the most trendy 'do. One rapper from Mexico had hooks implanted into his head so he could rock a full head of bling… and to be quite honest, it looks pretty sick. Trikafta won a big ol' prize for extending Jer's life! Thai food is delicious but one dish may kill you with one bite and for WHAT THE HELLTH!?! The boys cover an insanely shocking story of one woman who's blood was so toxic that it landed multiple healthcare providers in the hospital after trying to save her life! Join the post-episode conversation over on Discord! https://discord.gg/expeUDN

Bean Corcoran, and Will Corcoran.  Mother and son.I met Bean Corcoran when we were doing a science and innovation campaign, and we became friends!  I learned a little bit about her son Will, who has CF.  Bean is very involved in the CF community.  She is the President of the CFF Connecticut Chapter and the Bridge of Hope Communications Director volunteer.  We did a podcast in the past about the Bridge of Hope and what they do, so feel free to go back and listen to that if you want to find out more. She is also a Patient Family Partner and Quality Improvement Leader on the Pediatric CF Care team at Columbia Presbyterian Hospital in New York City since 2017. Bean is also an artist who paints endangered animals in oils and works in pottery. Bean and her husband live with their dog Rosie.Today we're talking about Bean and her son Will. Will cannot take the CF modulator, and we discuss what it does to you mentally and physically.  Will is 26-years old and lives in Chicago. Will was diagnosed with CF at 3 months old, received a liver transplant at age 15, and is unable to benefit from modulators due to his mutations. Will does four breathing treatments a day, rarely has more than two weeks without antibiotics, and battles hemoptysis. Will has been seen at five different CF centers. He is also an advocate. He works on family advisory boards, speaks with younger CFers, and currently sits on the Adult Advisory Council for the CFF. Will enjoys time outside with his dog Stanley, watching Philadelphia sports, and works as a fundraiser for an environmentally focused non-profit.I am grateful for Bean, (which is her nickname) and to her son Will for being so honest about how CF has impacted their lives.Please consider making a donation: https://thebonnellfoundation.org/donate/The Bonnell Foundation website: https://thebonnellfoundation.orgBonnell Foundation email: thebonnellfoundation@gmail.comThanks to our sponsors:Vertex:  https://www.vrtx.comGenentech: https://www.gene.comViatris: https://www.viatris.com/en

We'll be taking a break in May, June, and July while our host is on maternity leave. See you again in August!   This week on Pharm5: Vowst (SER-109) for recurrent C Diff Trikafta for CF in 2+ years Mounjaro for weight loss in diabetes Qalsody accelerated approval for ALS J&J talc baby powder settlement   Connect with us! Listen to our podcast: Pharm5 Follow us on Twitter: @LizHearnPharmD   References: FDA approves first orally administered fecal microbiota product for the prevention of recurrence of Clostridioides difficile infection. U.S. Food and Drug Administration. https://bit.ly/3Li7Cue. Published April 26, 2023. Accessed April 27, 2023. Feuerstadt P, Louie TJ, Lashner B, et al. Ser-109, an oral microbiome therapy for recurrent clostridioides difficile infection. New England Journal of Medicine. 2022;386(3):220-229. doi:10.1056/nejmoa2106516 Vowst [package insert]. Cambridge, MA: Seres Therapeutics, Inc.; 2023. FDA approves Trikafta for children ages 2 through 5 years with certain CF mutations. Cystic Fibrosis Foundation. https://bit.ly/44dP3QD. Published April 26, 2023. Accessed April 27, 2023. Lobo A. FDA expands its approval of Trikafta to cover younger children. Cystic Fibrosis News Today. https://bit.ly/3oSuhG0. Published April 27, 2023. Accessed April 27, 2023. CFTR modulator therapies. Cystic Fibrosis Foundation. https://bit.ly/3Az4epZ. Accessed April 27, 2023. Lilly's tirzepatide achieved up to 15.7% weight loss in adults with obesity or overweight and type 2 diabetes in surmount-2. Eli Lilly and Company. https://bit.ly/3VkD9AE. Published April 27, 2023. Accessed April 27, 2023. Galvez-Jiminez N, Goyal NA, Cudkowicz ME. Disease-modifying treatment of amyotrophic lateral sclerosis. UpToDate. http://www.utdol.com. Updated May 17, 2022. Accessed May 19, 2022. Liu A. FDA awards Biogen's ALS Drug Qalsody an accelerated approval, following its experts' feedback this time. Fierce Pharma. https://bit.ly/3VaVwrC. Published April 25, 2023. Accessed April 27, 2023. FDA approves Qalsody™ (Tofersen) as the first treatment targeting a genetic cause of ALS. PR Newswire: press release distribution, targeting, monitoring, and marketing. https://bit.ly/3ADSe6N. Published April 25, 2023. Accessed April 27, 2023. Qalsody [package insert]. Cambridge, MA: Biogen, Inc.; 2023. J&J to retain all talc-related liabilities from litigation in US, Canada. Reuters. https://bit.ly/3HjTS10. Published April 27, 2023. Accessed April 27, 2023. Spector M, Knauth D. J&J unit files for second bankruptcy to pursue $8.9 billion talc settlement. Reuters. https://bit.ly/426xfoY. Published April 5, 2023. Accessed April 27, 2023.

"Sorry kid, you're not sick enough for us anymore." This week the fellas discuss the Make-A-Wish Foundation's controversial decision to change their policy on cystic fibrosis. Apparently, medical advances have made it possible for these patients to live longer, which means they don't automatically qualify for a wish anymore. Also, did you know a year's worth of Trikafta could be made as cheap as $5700? Yet some poorer countries aren't being given access to the drug and it's killing young folks with CF as a result. Why are orcas killing sharks and removing their livers with surgical precision? Maybe Liver King was right, Liver is king. Get ready to yawn… seriously though, the guys probably yawned 8 times between them during the show. Finally for WHAT THE HELLTH!? Gym bros are eating dog food for gains. It's disgusting. Join the post-episode conversation over on Discord! https://discord.gg/expeUDN

For years, a diagnosis of cystic fibrosis has often meant an early death for patients. This week, Cystic Fibrosis Canada released its annual data report this week for 2021. A new drug, Trikafta, is keeping patients out of hospitals and off of transplant lists.The Globe's health reporter Kelly Grant explains why doctors are now talking about cystic fibrosis as a disease in transition and the hope of a new future for thousands of patients.Questions? Comments? Ideas? Email us at thedecibel@globeandmail.com

Zachariah Churchill is a Canadian MLA from Nova Scotia. He serves as a member of the Nova Scotia House of Assembly for Yarmouth. During his tenure as health minister, Zach committed to providing drug coverage for Trikafta, a life-saving drug for people with cystic fibrosis. In opposition, Zach advocated for this commitment to be followed through and was successful. Cystic fibrosis patients in Nova Scotia began receiving coverage on November 18, 2021. This week Zach joins the fellas to give them an inside look at what it means to be the health minister.

Our Angel Number Podcast! FGF 122 on July 22, 2022. This episode is all about alignment. The forecast today calls for a 10% chance rocket debris falling from the sky and killing you. Trikafta is now approved for kids 6-11 in Nova Scotia! The countries youngest recipient of an artificial heart is cute as a button. A sex toy a day keeps the doctor away... just gotta find a way around those pesky fun toy laws in Alabama. Uber drops the ball for disabled customers and pays the price. UPS trucks are a death trap. For WHAT THE HELLTH?! We talk about why a certain tree this time of year smells like cummy. Yummy or Yuck? Join the post-episode conversation over on Discord! https://discord.gg/expeUDN

COVID finally got Jer. Although it's the zeta cuck variant. Zero symptoms. Either that or Trikafta is unknowingly one of the world's most impressive COVID therapeutics... not really. No misinformation here. Brian is full of hot takes. Tune in to hear mushrooms speak & to see the actual size of a pregnant uterus. Toby is a little CF warrior. Literally. The kid lives for punching other children in the face. The U.S. is addicted to cremation. Short guys can now get the long end of the stick with a leg-lengthening surgery. It's all about confidence fellas! Finally in this week's edition of WHAT THE HELLTH!? One poor man injected insulation foam into a place where really you shouldn't be injecting anything. Spoiler alert it has to do with his Pee Pee and he will never be able to use it again! *Join the post-episode conversation over on Discord! https://discord.gg/expeUDN