Group of genetic blood disorders
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Gene therapy has changed what is possible for people living with sickle cell disease. New treatments have shown that the condition can be successfully treated. But the therapy is costly and available to only a small number of patients. Millions of people around the world still rely on early diagnosis and preventative medicines, with access to basic care remaining a challenge in many places, including sub-Saharan Africa, which has almost 80% of global sickle cell cases. Are we closer to gene therapy for sickle cell disease being accessible?We speak to Dr Natasha Archer, director of the Sickle Cell Program at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, US; Dr Alexis Thompson, paediatric haematologist at the Children's Hospital of Philadelphia and a professor of paediatrics at the University of Pennsylvania, US; Dr Leon Tshilolo, professor of paediatrics and haematology at the University of Mbuji-Mayi, DR Congo and Prof Baba Inusa, consultant paediatrician in the School of Life Course & Population Sciences at King's College London, UK(Photo: Cell lines are prepared in a laboratory in Maryland, US. Credit: Brendan Smialowski/Getty Images)Presenter: Daniel Rosney Producer: Matt Toulson Researcher: Evie Yabsley Sound engineer: Cameron Ward Production co-ordinator: Phoebe Lomas and Liam Morrey Editor: Tom Bigwood
Sickle cell is a genetic blood condition that can cause severe pain, extreme tiredness and serious health complications. It mainly affects people of African and Caribbean heritage, and for many, it's something they've had to live with their whole lives. But that could be changing.We meet Daniel Cressy, a young man from Louisiana whose dream is to become a pilot. After years of living with sickle cell, he received a ground-breaking treatment that has effectively cured him. He shares what life was like before and what it's like now to be sickle cell free.We'll also hear from Dr Steven Okoli, who breaks down how these new treatments work and what needs to happen to make them available to more people across the world. Could we be entering a future where sickle cell is no longer a lifelong condition? Email: whatintheworld@bbc.co.uk WhatsApp: +44 330 12 33 22 6 Presenter: Emma-Louise Amanshia Producers: William Lee Adams and Stefania Okereke Video Producer: Baldeep Chahal Editor: Verity Wilde
A college football player died because of a sickle cell disease and intense conditioning full 2447 Wed, 08 Jul 2026 04:00:00 +0000 947BscDGuHwHk2BehQaJut5wmIOVKQrY news MIDDAY with JAYME & WIER news A college football player died because of a sickle cell disease and intense conditioning From local news & politics, to what's trending, sports & personal stories...MIDDAY with JAYME & WIER will get you through the middle of your day! © 2025 Audacy, Inc. News https:
Dr. Z and Dr. C sit down with Dr. Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios Pharmaceuticals, to break down one of the most frequently heard but least understood terms in the treatment conversation: FDA approval. From the phases of clinical development and the role of regulators, to the difference between traditional and accelerated approval pathways, Dr. Gheuens demystifies the science and the process in plain language. They also discuss why asking questions of physicians about their care is one of the most important things a patient can do. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Hosts Drs. Ahmar Zaidi and Mike Callaghan are Agios employees.
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. The first quarter of 2026 has brought a wave of significant changes and advancements in the industry, marked by crucial regulatory updates, strategic acquisitions, and shifts in the competitive landscape. A pressing issue for many companies is the looming patent cliff for several branded biologics. This situation presents both challenges and opportunities for biosimilars in the U.S. market. The introduction of biosimilars for major products like AbbVie's Humira, Johnson & Johnson's Stelara, and Regeneron/Bayer's Eylea is shifting the narrative from slow adoption to a more competitive biosimilar market landscape. Vertex Pharmaceuticals has achieved a milestone with the FDA expanding the label for its gene therapy Casgevy, now approved for treating children as young as two with sickle cell disease or transfusion-dependent beta thalassemia. This expansion emphasizes the potential of gene therapies to revolutionize treatment options for younger patients suffering from these conditions. In a pivotal development, Novartis announced a significant acquisition of UK biotech Myricx. With an upfront payment of $1.1 billion and potential total value up to $1.5 billion, this acquisition is set to enhance Novartis' antibody-drug conjugate (ADC) pipeline by incorporating Myricx's novel payload technology and NMT inhibitor payloads. This move reflects Novartis' focus on precision oncology by improving ADC efficacy through targeting critical enzymatic pathways involved in tumorigenesis. Amgen faced a setback with a voluntary recall of approximately one million bottles of its heart medication Corlanor due to potential contamination, highlighting the critical importance of maintaining rigorous manufacturing quality control to ensure drug safety. The regulatory landscape continues to evolve, illustrated by Robert F. Kennedy Jr.'s Department of Health and Human Services revoking lingering COVID-19 emergency authorizations. This signals a transition towards post-pandemic normalcy and an evolving focus on pandemic preparedness frameworks. Sanofi's Genzyme unit in Ireland faced criticism after an FDA inspection linked to Altuviiio resulted in a Form 483 notice. This incident underscores ongoing challenges in maintaining compliance with stringent regulatory standards and emphasizes the importance of robust quality assurance mechanisms. Lonza is expanding its capacity for producing ADCs and enhancing its partnership with a large U.S. drugmaker, aligning with industry trends toward outsourcing complex biologics manufacturing amid growing demand for ADCs. Genentech is undergoing restructuring, resulting in 103 layoffs as part of a strategic rethink of its early development group. Despite these changes, Genentech entered a $490 million collaboration focused on breast cancer, demonstrating its commitment to innovation amid organizational shifts. AstraZeneca continued its collaborative efforts by signing a $1.7 billion kidney drug discovery pact with Chinese biotech CSPC Pharmaceutical. This partnership emphasizes AstraZeneca's strategy to leverage global collaborations to accelerate drug discovery and development efforts. Scribe Therapeutics filed for an Initial Public Offering (IPO) to advance its CRISPR-based gene editing technologies targeting cardiovascular diseases at a genetic level, highlighting an ongoing shift toward personalized medicine. Celea Therapeutics raised $180 million in venture funding to advance Deupirfenidone into late-stage clinical trials for Idiopathic Pulmonary Fibrosis (IPF), signaling confidence in its therapeutic potential amid ongoing investment trends targeting rare diseases. The Medicines and Healthcare products Regulatory Agency expanded approval for Novo Nordisk's Wegovy for Metabolic-Associated Steatohepatitis with moderate-to-advanced liver fibrosis. This expansion represents significant progress given MASH's rising prevalence and progression risks. BridgeBio's ATTRibute-CM study yielded promising results with Acoramidis offering kidney-protective benefits in transthyretin amyloidosis cardiomyopathy patients—a potential new approach managing this rare disease. Revolution Medicines reported encouraging data for its KRAS inhibitor Zoldonrasib in combination therapies targeting metastatic pancreatic ductal adenocarcinoma—offering new strategies against aggressive cancer types. Brii Bio's phase 2b trial results show promise for a combination therapy aimed at achieving a functional cure for chronic Hepatitis B Virus infection—offering hope for millions affected by this condition. Robust fundraising activities continue within biotech sectors as Monash University and Atisama Therapeutics secure funding from MRFF grants developing therapies targeting chronic lung diseases. Overall, these developments highlight dynamic transformations reshaping therapeutic landscapes through precision medicine approaches leveraging cutting-edge technologies promising improved patient care across various domains within pharmaceutical industries globally.Support the show
9 Simple Solutions to Achieve Health Equity: A Guide for Healthcare Professionals and Patients by Mauvareen Beverley https://www.amazon.com/dp/B0D73NNN2V Drbeverley.com Nine Simple Solutions to Achieve Health Equity: A Guide for Healthcare Professionals and Patients by Dr. Beverley presents actionable strategies to transform health disparity into health equity. Drawing from her extensive interactions with over 1,000 diverse patients, Dr. Beverley introduces innovative approaches, like “The Bridge Team,” to enhance outcomes for vulnerable populations. Dr. Beverley offers a clear roadmap supported by real-life stories, addressing the critical gap between patient perception and reality. She emphasizes simple, empathetic solutions to prevent complex health issues, stressing the importance of recognizing patients as multifaceted individuals with roles beyond their medical conditions. This essential guide advocates for cultural competence across all patient demographics, highlighting the often-overlooked needs of the English-speaking Black population who are not included in the need for cultural competence. Dr. Beverley also provides historical insights into the health crises faced by Black communities, underscoring the need for equitable, compassionate care inclusive of individuals with Sickle Cell Disease. Echoing Hippocrates, Dr. Beverley reminds us: “It is more important to know the person who has the disease than the disease who has the person.” This book is a vital resource for healthcare professionals and patients committed to achieving health equity and improving health outcomes. About the author Mauvareen Beverley, M.D. is the President of Mauvareen Beverley, M.D., PLLC, Patient Engagement and Cultural Competence Specialist. She is an executive-level physician and a fellow of the New York Academy of Medicine (NYAM) with over twenty years of experience advocating for improving patient engagement and cultural competence for all populations, especially African American communities. As Assistant Vice President, Physician Advisor for NYC Health + Hospitals, she sponsored the first Conference on Improving the Health of the Elderly Black Population. Under her leadership as Deputy Executive Director of Kings County Hospital, she led her team to implement innovative strategies for improved equitable health outcomes and decreased Congestive Heart Failure readmission from 30 percent to 18.7 percent in less than two years. Dr. Beverley is a national thought leader and expert in patient engagement and health equity. She has lectured extensively on health disparities, patient engagement and valuing the human experience.
As Christians, we need to stay informed about the rapidly changing world of fertility treatments, genetic testing, and IVF before making decisions or forming opinions. Join C.L. as we explore the powerful story behind the Boozer family, wrestle with difficult questions about life, embryos, technology, and God's will, and seek biblical wisdom for navigating one of the most important ethical conversations of our time.
To commemorate World Sickle Cell Day 2026, the Cheat Codes podcast is re-releasing this powerful live episode from the 2025 National Alliance of Sickle Cell Centers (NASCC) meeting. In this episode, Dr. Z and Dr. C are joined by a powerhouse panel: Dr. Payal Desai, Dr. Seethal Jacob, Dr. Pat McGann, and Dr. Andy Ellner to unpack what truly defines great sickle cell care. It's a topic well in line with the theme of this year's World Sickle Cell Day, "Closing the Survival Gap: Equity in Sickle Cell Care." SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. With each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram.
What does good care actually look like for adults living with sickle cell disease?In this episode of our What Good Care Looks Like for Adults with Sickle Cell series, lifespan sickle cell expert Dr. Julie Kanter focuses on navigating treatment options and disease modifiers. Dr. Kanter breaks down the most prominent medications and therapies available today, including hydroxyurea, crizanlizumab, L-glutamine, and blood transfusions, explaining how patients and providers can work together to personalize care and find the best treatment path.Dr. Julie Kanter is the Co-Director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama at Birmingham and President of the National Alliance of Sickle Cell Centers (NASCC).This episode is part of Sickle Cell 101's Care and Treatment 101 Educational Initiative, a community resource dedicated to making care information accessible and actionable for the sickle cell community.Thank you to our Care and Treatment 101 sponsors: Vertex, Chiesi, Pfizer, and Medunik.
What does good care actually look like for adults living with sickle cell disease?In this episode of our What Good Care Looks Like for Adults with Sickle Cell series, lifespan sickle cell expert Dr. Julie Kanter focuses on the evolving landscape of sickle cell research and data collection. Dr. Kanter walks through the latest drug development approaches, such as fetal hemoglobin induction and reducing cell adhesion, while highlighting why safe data sharing through national registries is absolutely essential to advancing personalized treatments for the community.Dr. Julie Kanter is the Co-Director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama at Birmingham and President of the National Alliance of Sickle Cell Centers (NASCC).This episode is part of Sickle Cell 101's Research 101 Educational Initiative, a community resource dedicated to making care information accessible and actionable for the sickle cell community.Thank you to our Research 101 sponsors: Agios, Pfizer, and Beam Therapeutics.
Send us Fan MailIn this episode, we discuss how to identify and manage the acute complications of sickle cell disease. Written by Dr. Yi Hui Luo (Internal Medicine Resident). Reviewed by Dr. Véronique Naessens (Hematologist) and Dr. Sanabelle Zaabat (General Internist). Support the show
What does good care actually look like for adults living with sickle cell disease? In this episode of our What Good Care Looks Like for Adults with Sickle Cell series, lifespan sickle cell expert Dr. Julie Kanter focuses on two pillars of quality sickle cell care: working with the right specialists and understanding the lab work that guides your treatment.Dr. Julie Kanter is the Co-Director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama at Birmingham and President of the National Alliance of Sickle Cell Centers (NASCC).This episode is part of Sickle Cell 101's Care and Treatment 101 Educational Initiative, a community resource dedicated to making care information accessible and actionable for the sickle cell community.Thank you to our Care and Treatment 101 sponsors: Vertex, Chiesi, Pfizer, and Medunik.
What does good care actually look like for adults living with sickle cell disease? In this episode of our What Good Care Looks Like for Adults with Sickle Cell series, lifespan sickle cell expert Dr. Julie Kanter breaks it down. She covers establishing a medical home, working with a sickle cell specialist, navigating the Emergency Department, and building an Individualized Care Plan that works for you.Dr. Julie Kanter is the Co-Director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama at Birmingham and President of the National Alliance of Sickle Cell Centers (NASCC).This episode is part of Sickle Cell 101's Care and Treatment 101 Educational Initiative, a community resource dedicated to making care information accessible and actionable for the sickle cell community.Thank you to our Care and Treatment 101 sponsors: Vertex, Chiesi, Pfizer, and Medunik.
This week on the Lin. Woods Gospel Entertainment Podcast, we're revisiting an inspiring conversation with rising gospel artist Preston Jonzell, a powerful voice with an even more powerful testimony.Preston shares his incredible journey from being known as "the kid with the high voice" to becoming one of gospel music's most exciting emerging artists. He opens up about the inspiration behind his hit single "Big God," his songwriting process, and how his faith has sustained him through one of life's greatest challenges, living with Sickle Cell Disease.This heartfelt and uplifting conversation is filled with hope, perseverance, faith, and the reminder that no matter what you're facing, we serve a BIG GOD!
Sickle cell disease causes accelerated kidney function decline, yet proven GFR-preserving therapies remain elusive. In this study of adults with sickle cell disease, RASi use was not associated with a significant difference in the rate of eGFR decline.
Hematological emergencies though uncommon can be a significant problem and is something we should all be familiar with. That is why I asked Dr. Kenneth Bujold (previous intern of mine) to come on the show and tell us how can we do a better job handling true emergencies in Sickle Cell Disease patients.
May 8, 2026- Legislation has been kicking around the Capitol for more than a decade that would promote more specialized care for New Yorkers with sickle cell disease. We discuss this effort with Ginger Davis, president of the Sickle Cell Thalassemia Patients Network.
Toni Eyssallenne, Youri Louis, and Pierre Minn join us to discuss the larger structural, social, and historical forces which shape healthcare in Haiti. Rather than treating health disparities as isolated or purely clinical problems, this conversation situates them within Haiti's political economy, colonial and revolutionary legacy, and ongoing global relationships around debt and power. The discussion traces how structural forces shape disease patterns, the delivery of care, and experience as patient and clinician.This episode is a collaboration with the Campaign Against Racism (CAR) and the CAR-Haiti chapter; enormous thanks to CAR's Anne Marie Collins and Youri Encelotti Louis.Resources:"Where They Need Me" by Pierre Minn (2022)St. Damien HospitalHelp! Make it Make Sense podcast (cohost Toni Eyssallenne)SocMed Alumni HaitiGuests:Toni Eyssallenne graduated from the University of Rochester School of Medicine and Dentistry's Medical Scientist Training Program (MSTP). Following her chief residency of the combined Internal Medicine-Pediatrics residency program at Jackson Memorial Hospital in Miami, Florida, she worked as a leader in clinical and academic medicine in both the US and Haiti. She designed and led clinical and academic tracks, programs, and departments for faculty, trainees and students in both countries and has dedicated her professional career to underserved communities domestically and abroad. She is currently the Deputy Chief Medical Officer at the NYC Department of Health and Mental Hygiene and continues to provide primary care to both adults and children with Strong Children Wellness in Queens, NY. Her scholarly work focuses on Sickle Cell Disease and Anti-Racism in clinical medicine and medical education.Pierre Minn is Associate Professor in the Department of Anthropology at the Université de Montréal. His research interests include the moral dimensions of medical practice, transnational humanitarian aid, and global health education and practice. He has conducted long-term ethnographic fieldwork in Haiti since the late 1990s. He is the author of Where They Need Me: Local Clinicians and the Workings of Global Health in Haiti (Cornell University Press, 2022), which analyzes the place of Haitian clinicians the context of foreign medical aid and development projects.Youri Encelotti Louis is a Haitian-trained physician and social medicine expert dedicated to dismantling systemic healthcare inequities. As a Co-Founder of SocMed Alumni Haiti, Board member - Program Lead at EqualHealth and Care Coordinator at Dialogue Health, he leverages over a decade of experience in program management and research to advance decolonization and health equity. His work centers on building community-led initiatives that bridge the gap between medical practice and social justice.
Nabeelah Peerbhai shares her lived experience with SIckle Cell Disease and how her pain has led to her purpose in Genetic Counselling. Join us to learn more about sickle cell and genetic counselling.Join us on the 23 April 2026 at 12pm CAT on www.vukaonlineradio.co.za as we have a chat with our host Colleen and special guest Nabeelah Peerbhai.Be sure to join us! #Vuka #Healthcare #Medical
Transformative therapies for sickle cell disease are redefining what is possible for patients, offering the potential for cure alongside substantial risks. In CMAJ, the article Transformative therapies for sickle cell disease outlines how stem cell transplant and emerging gene therapies are changing the trajectory of a condition long defined by recurrent crises, shortened life expectancy, and inequities in care.Dr. Kareem Jamani, a haematologist and clinical associate professor at the University of Calgary, explains how stem cell transplant replaces a patient's blood-forming system to eliminate sickling haemoglobin, offering what can reasonably be considered a cure. Outcomes are generally favourable, particularly with matched sibling donors, but risks remain, including graft-versus-host disease, infertility, rejection, and mortality that can reach 5–7% with less well-matched donors. He also outlines the role of gene therapy, which modifies a patient's own stem cells to increase fetal haemoglobin production, resulting in a functional cure with similar improvements in quality of life.The episode is grounded in the experience of Ufuoma Muwhen, who underwent a stem cell transplant as a teenager after years of frequent hospitalizations and functional limitations. She describes the toll of both the disease and the treatment process, as well as the shift in her daily life following transplant, including her ability to travel, exercise, and live without recurrent crises. Her account highlights the importance of wraparound supports and trust in the healthcare system.For physicians, these therapies require balancing meaningful benefit against real risk, often early in life and under uncertainty. The discussion also underscores persistent inequities, including delayed acute care, limited access to fertility preservation, and the challenges of delivering advanced therapies within systems designed around oncology rather than chronic genetic disease.For more information from our sponsor, go to medicuspensionplan.comComments or questions? Text us.Join us as we explore medical solutions that address the urgent need to change healthcare. Reach out to us about this or any episode you hear. Or tell us about something you'd like to hear on the leading Canadian medical podcast.You can find Blair and Mojola on X @BlairBigham and @DrmojolaomoleX (in English): @CMAJ X (en français): @JAMC FacebookInstagram: @CMAJ.ca The CMAJ Podcast is produced by PodCraft Productions
In this powerful episode of Cheat Codes, Dr. Z and Dr. C sit down with Dr. Santosh Saraf, one of the leading experts on kidney disease in sickle cell, to break down why renal complications are so common yet often overlooked. Dr. Saraf walks listeners through the early warning signs, the importance of routine monitoring, and how albuminuria, creatinine, and cystatin C can signal kidney stress long before symptoms appear. The discussion explores how anemia, hemolysis, blood pressure, and even treatment choices influence long-term kidney health, along with emerging therapies that offer new hope for protection and repair. This is an essential conversation for anyone living with sickle cell disease or caring for someone who is. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Hosts and guest featured in this episode have been compensated for their time.
This episode covers sickle cell disease.Notes: https://zerotofinals.com/paediatrics/haematology/sicklecell/Questions: https://members.zerotofinals.com/Books: https://zerotofinals.com/books/The audio in the episode was expertly edited by Harry Watchman.
In this episode of Hema Now, Karina Yazdanbakhsh speaks about her journey into immunology, the challenges of alloimmunisation, and the evolving landscape of transfusion medicine. She also reflects on receiving the prestigious Henry M. Stratton Medal for Basic Research at ASH 2025 and what this recognition means for her work. The conversation explores key insights from ASH 2025, highlights pressing immunological issues in sickle cell disease, and looks ahead to emerging technologies shaping personalised transfusion strategies. Timestamps: 00:00 – Introduction 00:59 - Stratton Medal meaning 02:19 - ASH 2025 highlights 13:11 - Sickle cell challenges 17:00 - Immune tolerance evolution 18:55 - Institute research priorities 27:20 - Novel therapeutics role 29:04 - Early career advice 29:43 - Emerging technologies impact 32:30 – Three magic wishes for healthcare
This PedsCases episode will review the Canadian Paediatric Society Position Statement from November 2022, "Acute complications in children with sickle cell disease: Prevention and management". By the end of this podcast, listeners will be able to 1) understand how to prevent mortality and morbidity associated with SCD, 2) recognize and manage the common complications of SCD, and 3) know the basic principles of transfusion for patients with SCD. Today's episode was created by Audrey Slater, a fourth-year medical student at the Université de Montréal, in collaboration with the authors of the CPS statement, including Dr. Carolyn E. Beck, a hospitalist pediatrician at SickKids hospital, Dr. Evelyne D. Trottier, a pediatric emergency physician at CHUSJ, Dr. Melanie Kirby-Allen, a haematologist at SickKids Hospital, and Dr. Yves Pastore, a haematologist at CHUSJ. There are no conflicts of interest to disclose by the authors.
In this podcast, Baba delves into his expertise in hematology, sharing what inspired his journey into this field of research. He also discusses the Academy for Sickle Cell and Thalassaemia (ASCAT) Conference, which he founded to unite experts and professionals working to tackle hematological disorders like sickle cell disease and thalassemia.
In this episode, Sonika and Gabe explore how pain is experienced differently across bodies and interpreted differently in conversations, with a focus on people living with Sickle Cell Disease. This conversation dips into acute and chronic pain, highlighting the challenge health communicators face in bringing the unseen aspects of someone's pain to light. This episode was recorded in October 2025.Follow us on LinkedIn
In this episode of Cheat Codes, Dr. Z and Dr. C sit down with Dr. Nirmish Shah of Duke University for a deep dive into how technology, wearables, and patient-reported outcomes are transforming the way clinicians measure quality of life in sickle cell disease. Dr. Shah shares the evolution of his work tracking daily symptoms, passive data, and long-term trends, and explains how these insights can help predict complications, personalize care, and strengthen the patient–provider relationship. The conversation explores everything from AI-driven analytics to hospital-based monitoring, highlighting how data can support—not replace—the human side of medicine. This episode offers a fascinating look at the future of sickle cell care and the innovations shaping it today. To hear the episode with Dr. Charles Jonassiant and Jude Jonassiant referenced in the episode, CLICK HERE. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Hosts and guest featured in this episode have been compensated for their time.
On this episode host Fred Goldstein invites Angela Luong, PharmD, Senior Clinical Consultant at Pharmaceutical Strategies Group in this last installment of our rare disease series. Our discussion focuses on the burden of sickle cell disease, chronic pain management, limited treatment options, and the implications for managed care programs, including the use of data and care coordination to improve patient outcomes. This activity is supported by an independent medical education grant from Agios Pharmaceuticals, Inc. AMCP offers CPE for this podcast through December 31, 2026. For additional information and to claim credit, please visit: The Power of Partnership: Bridging Patients and Payers in Sickle Cell Disease Management. Find all of our network podcasts on your favorite podcast platforms and be sure to subscribe and like us. Learn more at www.healthcarenowradio.com/listen/
At 3 months old, Victoria Gray wouldn't stop crying. Blood tests brought devastating news: she had sickle cell disease, a genetic blood disorder that blocks blood flow and oxygen delivery to the body. It causes unbearable pain that Victoria describes as “getting struck by lightning and hit by a truck.”As she got older, Victoria felt increasingly isolated and hopeless. She often spent her kids' birthdays at the hospital, where she received regular blood transfusions. “I felt like I was cheating my children out of their childhood,” she says. “I didn't look forward to a long life. I stopped dreaming. I gave up on school or doing anything … I thought that I was close to dying.”But at age 34, Victoria got a new chance at life. In 2019, she became the first person in the world to receive a revolutionary new treatment for the disease — a gene-editing tool called CRISPR discovered in a UC Berkeley lab, which would go on to win a Nobel Prize just one year later. “It felt like an answered prayer for me,” says Victoria. “CRISPR not only freed me, it freed my children.” This is the third episode of our latest Berkeley Voices season, featuring UC Berkeley scholars working on life-changing research — and the people whose lives are changed by it.Listen to the episode and read the transcript on UC Berkeley News (news.berkeley.edu/podcasts/berkeley-voices).Music by Blue Dot Sessions.Photo courtesy of Victoria Gray; illustration by Neil Freese/UC Berkeley. Hosted on Acast. See acast.com/privacy for more information.
In this episode of Cheat Codes, Dr. Sharl Azar joins the hosts to discuss his journey from Oregon to leading the sickle cell program at Mass General in Boston. Dr. Azar shares the pivotal moments that inspired his passion for hematology and advocacy, including his work on groundbreaking legislation to improve care for sickle cell patients in Massachusetts. The conversation highlights the importance of community, systemic change, and the lived experiences of patients. Listeners will be inspired by Dr. Azar's commitment to equity, collaboration, and building a brighter future for those affected by sickle cell disease. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
In today's episode, we're addressing an often-overlooked topic in dentistry—managing dental patients with Sickle Cell Disease and Sickle Cell Trait. These patients face unique challenges, and unfortunately, many are turned away from dental practices simply because their condition isn't well understood. Our guest, Linda Chandler, RDH, s a graduate of the University of Texas School of Dental Hygiene, past President of the Southeast National Dental Hygiene Association, author, educator and voted top hygienist of the year 2012.
Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the intricate tapestry of scientific advancements, regulatory decisions, and strategic maneuvers shaping our industry.One of the notable stories involves Agios Pharmaceuticals, which is pushing forward with its sickle cell disease treatment, Pyrukynd, for FDA approval. This comes despite mixed results from their Phase 3 clinical trials, which led to a significant drop in their stock value. This scenario underscores the complexities of navigating clinical trial outcomes while pursuing breakthroughs in treating challenging diseases like sickle cell.Arrowhead Pharmaceuticals has marked a significant milestone with the FDA's approval of Plozasiran. This achievement not only marks Arrowhead's entry into the commercial sector but also highlights the competitive dynamics within biotech, as companies like Ionis Pharmaceuticals vie for market dominance with innovative therapies. Further strengthening its position, Arrowhead also received FDA approval for Redemplo, a siRNA-based therapeutic for rare genetic metabolic disorders. Despite facing volatility due to safety concerns in its partnership with Sarepta Therapeutics, this approval underscores RNA interference therapies' potential in precision medicine.In corporate strategy news, Alkermes is making moves to acquire Avadel Pharmaceuticals, offering up to $2.37 billion and overshadowing a competing bid from Lundbeck. Such acquisitions are part of a broader trend of consolidation in the industry aimed at expanding portfolios and market reach. Avadel's decision to accept Alkermes' revised offer over Lundbeck's bid highlights ongoing consolidation trends as companies expand their portfolios in competitive markets like narcolepsy drugs.On the investment front, Celltrion has committed $478 million to upgrade a U.S. manufacturing facility acquired from Eli Lilly. This expansion is crucial for increasing manufacturing capabilities within the biosimilars sector, where demand for cost-effective therapeutics is on the rise. Additionally, Celltrion's exploration beyond biosimilars with a potential $350 million deal involving Trioar's antibody platform demonstrates ambition to diversify its portfolio towards innovative biologics.Teva Pharmaceuticals is fostering innovation by inviting startups to tackle key challenges within biopharma through a global platform. This initiative reflects a growing trend toward open innovation and collaboration, seeking novel solutions to complex issues across research and development and manufacturing efficiencies.In regulatory news, the controversial $7.4 billion settlement plan involving the Sackler family and Purdue Pharma has received approval from a bankruptcy judge. This paves the way for Purdue's transformation into Knoa Pharma and highlights ongoing legal and ethical reckonings related to opioid liabilities within the industry.Cytokinetics remains committed to its independent path as it awaits FDA approval for its cardiovascular drug Aficamtem. The company's determination to commercialize without big pharma support reflects a trend where smaller biotech firms strive for autonomy while bringing first-in-class drugs to market.On an infectious disease front, Merck has demonstrated significant progress with its HIV treatment Islatravir in Phase 3 trials. This places Islatravir as a potential competitor against Gilead's Biktarvy, showcasing ongoing innovation within antiviral drug development.Additionally, Dexcom has gained clearance for its type 2 diabetes software integrating continuous glucose monitoring technology. This advancement exemplifies how digital health technologies are transforming chronic disease management bySupport the show
On this episode, Dr. Z and Dr. C welcome Shayan Siddiqui, founder of MiCaringHeart, to discuss his impactful work supporting sickle cell patients in Detroit. Shayan shares how his family's experiences inspired initiatives like Cabs for Kids, providing transportation to medical appointments, and Outsmart, offering free tutoring for children with chronic illnesses. The conversation highlights the challenges of healthcare access and the power of community-driven solutions. For more information about MiCaringHeart, visit https://www.micaringheart.com/ SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
On this week's Lin. Woods Gospel Entertainment Podcast, Lin. sits down with rising gospel star Preston Jonzell , a powerful voice with a testimony to match.Preston opens up about his musical journey, from being “the kid with the high voice” to becoming one of gospel music's most exciting new artists. He shares the inspiration behind his hit single “Big God,” his songwriting process, and how faith sustains him through life's biggest challenge , living with Sickle Cell Disease.This heartfelt conversation is full of hope, resilience, and the reminder that we truly serve a Big God! Don't miss it — tune in now on Podomatic, Apple Podcasts, Spotify, Audible, or wherever you listen to podcasts.Follow Lin. Woods: Instagram & Tik TOk @linwoods96 Facebook & LinkedIn @Lin. Woods X (Twitter) @LinWoods
In this special episode of Cheat Codes, Dr. Z and Dr. C hand the co-hosting mic to Dr. Julie Kanter for a lively walk through the history of the National Alliance of Sickle Cell Centers (NASCC) annual meetings. Alongside Dr. Sophie Lanzkron, Dr. Francis Coyne, and Dr. Alexis Leonard, they revisit key moments from 2022's belly-dancing dinner to 2025's record-breaking Vermont gathering. The conversation covers how NASCC consensus topics like pain management, iron overload, gene therapy readiness, and defining a "sickle cell specialist" have evolved year-to-year. Packed with behind-the-scenes stories, professional insights, and a look at NASCC's growing impact, this episode captures how a community of experts is shaping the future of sickle cell care. For more information on the National Alliance of Sickle Cell Centers (NASCC), please visit: https://sicklecellcenters.org/ SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
In this Rising Star episode from the NASCC meeting in Stowe, Vermont, Dr. Z and Dr. C spotlight Dr. Alyssa Schlenz, a psychologist transforming sickle cell care through early developmental screening and neuropsychological support. Dr. Schlenz shares her journey into the field, the critical role of prevention and early intervention, and the unique challenges faced by patients with neurodevelopmental disorders. She explains how routine, relationship-based screenings can identify issues early, reduce stigma, and improve long-term outcomes. This inspiring conversation highlights the power of psychology in comprehensive sickle cell care and the promising future being shaped by dedicated new leaders. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram.
Eberechi Nwogu-Onyemkpa is an assistant professor in the Division of Palliative Medicine at Washington University in St. Louis. Stephen Morrissey, the interviewer, is the Executive Managing Editor of the Journal. E. Nwogu-Onyemkpa and Others. Involving Palliative Care to Improve Outcomes in Sickle Cell Disease. N Engl J Med 2025;393:1553-1556. E. Costa and Others. Thirty Years of Hydroxyurea for Sickle Cell Anemia — Scientific Progress, Global Health Gaps. N Engl J Med 2025;393:1556-1559.
In this live episode from the NASCC meeting in Stowe, Vermont, Dr. Z and Dr. C are joined by a powerhouse panel: Dr. Payel Desai, Dr. Seethal Jacob, Dr. Pat McGann, and Dr. Andy Ellner to unpack what truly defines great sickle cell care. The group explores the importance of comprehensive, continuous, and patient-centered models, from inpatient to outpatient settings, pediatrics to adulthood. They discuss integrating technology, clinical trials, and multidisciplinary teams to improve quality of life and prevent long-term complications. It's an insightful look at how collaboration, innovation, and trust are shaping the future of sickle cell treatment. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. With each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
Sickle cell disease was once considered an incurable disease, until recently. A wide variety of gene therapies have now become available, allowing patients with sickle cell disease to now have futures once thought unimaginable. In this podcast episode, we talk through these treatments and get expert advice on what these treatments mean for our future.
As Sickle Cell Awareness Month comes to a close, we revisit our conversation with Tahirah Austin-Muhammad, who was diagnosed with sickle cell disease when she was six years old. Throughout her life, she has dealt with pain and organ failures. In 2024, she was on the waiting list for a kidney transplant, living on 7% kidney function. Despite that, she has raised a daughter and helped found Crescent Foundation to provide education and resources to others living with sickle cell disease. Racquel Williams talks with Tahirah along with her physician, Dr. Safiyya Shabazz. Learn more about how to find and support Black physicians in the Philadelphia area from The Medical Society of Eastern Pennsylvania. Then, on Shara in the City, we're treating ourselves to a little something sweet at Reading Terminal Market. Shara Dae Howard visits Sweet Nina's to learn about the local business and taste their wide array of banana pudding flavors. The sickle cell interview was originally released on May 18, 2024. Shara in the City at Sweet Nina's was originally released on August 2, 2025. To learn more about listener data and our privacy practices visit: https://www.audacyinc.com/privacy-policy Learn more about your ad choices. Visit https://podcastchoices.com/adchoices
In this episode of Cheat Codes, Dr. Z and Dr. C sit down with Dr. Julie Kanter, Dr. Sophie Lanzkron, and Dr. Marsha Treadwell to spotlight the National Alliance of Sickle Cell Centers (NASCC). They explore how NASCC came to life, its mission to unite multidisciplinary teams, and its role in setting consensus guidelines for sickle cell care. From building a national registry to fostering collaboration between clinics, community organizations, and patients, the conversation captures a pivotal movement toward consistent, high-quality care nationwide. Listeners will hear how NASCC is shaping the present—and future—of sickle cell treatment. For more information on the National Alliance of Sickle Cell Centers (NASCC), please visit: https://sicklecellcenters.org/ SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
Discover the powerful history of Trinidad as Diane Hinds, entertainment publicist and author of Conquerabia: The Struggle for Identity, unpacks the island's colonial past, the fight for identity, and the enduring impact of slavery and resistance. Learn how historical fiction can reshape narratives and why reclaiming lost voices is more important than ever. This insightful conversation is a must-watch for history lovers, writers, and cultural enthusiasts.==========================================
Combating the Opioid crisis in the capital city. Fentanyl Harm Prevention Kits and Emergency Overdose Boxes are now in the hands of first responders.Then, a new online tool is launched to address Mississippi's childcare shortages. Plus, Sickle Cell disease causes severe pain and even death. Efforts are underway to raise awareness about the disease and find a cure. Hosted on Acast. See acast.com/privacy for more information.
In this episode of Cheat Codes, Dr. Z and Dr. C are joined by renowned advocates TaLana Hughes and Cassandra Trimnell to explore what “the sickle cell community” truly means. They discuss how patients, caregivers, providers, and allies form interconnected, and often overlapping, communities that bring hope, support, and change. From building trust to celebrating wins, the conversation highlights the power of connection, the importance of access to information, and how to find your “tribe” in the sickle cell space. Whether local or global, online or in person, community remains the heartbeat of progress in sickle cell disease. SHOW DESCRIPTION Cheat Codes is intended for patients, caregivers, providers, and the greater community of people who are impacted by Sickle Cell Disease. Each episode, Cheat Codes strives to provide listeners with critical education, the latest scientific updates, and voices from the Sickle Cell community. Join an inclusive community and build connections with other hemolytic anemia allies by following @AllyVoicesRising on Instagram. TRANSPARENCY STATEMENT Cheat Codes: A Sickle Cell Podcast is made possible by Agios Pharmaceuticals Inc. Visit Agios.com to learn more. The following Agios-supported programs are intended for informational and educational purposes only and are not intended as medical advice. Please speak with your healthcare professional before making any treatment decisions. Host and guests featured in this episode have been compensated for their time.
a focus on its acute presentations and the care we can deliver to improve outcomes for our patients. Sickle cell disease (SCD) is a lifelong inherited blood disorder that affects over 15,000 people in the UK, and millions worldwide. It's caused by the production of abnormal haemoglobin molecules, which distort red blood cells into a crescent, or “sickle,” shape. These rigid cells can block small blood vessels, leading to painful vaso-occlusive crises and organ damage. While the condition has long been most prevalent in parts of Africa, the Middle East, the Mediterranean and India, today it's a global health issue, and one we encounter regularly in UK emergency care. Tragically, failings in care have too often led to avoidable harm. The 2021 parliamentary report “No One's Listening” laid bare some of these cases, highlighting missed opportunities, poor awareness, and systemic issues that cost lives, such as the death of Evan Nathan Smith. So why are we revisiting this now? In 2024, RCEM published new Best Practice Guidelines on managing sickle cell disease in the ED. These provide clear, evidence-based standards for recognition, triage, analgesia, infection control, and safe discharge. In this episode, we take you through the key elements; Pathophysiology – how a genetic mutation drives sickling, vaso-occlusion and inflammation. Clinical presentations – from painful crises and acute chest syndrome, to stroke, anaemia, infection, priapism and pregnancy-related complications. Recognition and triage – why timely pain control within 30 minutes is a must, and how to spot red flags. Investigations and treatment – including the role of reticulocytes, the importance of knowing a patient's baseline haemoglobin, and principles of analgesia, transfusion, oxygen, and supportive care. Discharge and ongoing care – ensuring safe, joined-up planning, and involving haematology and specialist pathways wherever possible. The take-home message? Every sickle cell crisis is a medical emergency. We need to listen to patients, escalate early, involve haematology, and deliver care that meets the standards they deserve. Once again we'd love to hear any thoughts or feedback either on the website or via X @TheResusRoom! Simon, Rob & James
About this episode: Sickle cell disease affects an estimated 100,000 people in the United States. Recent advancements in gene therapies and medicines like hydroxyurea are diminishing extreme pain, reducing strokes, and extending survival times for those afflicted by the disease. In this episode: leading sickle cell disease expert Dr. Mark Gladwin explains how revolutionary new treatments work and discusses the challenges to access to life-saving care. Guest: Dr. Mark Gladwin is a physician-scientist and the Dean of the University of Maryland School of Medicine and Vice President for Medical Affairs at the University of Maryland, Baltimore. His research focuses include sickle cell disease and hypertension. Host: Dr. Josh Sharfstein is vice dean for public health practice and community engagement at the Johns Hopkins Bloomberg School of Public Health, a faculty member in health policy, a pediatrician, and former secretary of Maryland's Health Department. Show links and related content: New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle—CNBC Gene Therapy: What You Need to Know—Sickle Cell Disease Association of American No More Pain: Breakthrough Sickle Cell Treatment from Johns Hopkins Offers Curative Potential—Johns Hopkins School of Medicine Transcript information: Looking for episode transcripts? Open our podcast on the Apple Podcasts app (desktop or mobile) or the Spotify mobile app to access an auto-generated transcript of any episode. Closed captioning is also available for every episode on our YouTube channel. Contact us: Have a question about something you heard? Looking for a transcript? Want to suggest a topic or guest? Contact us via email or visit our website. Follow us: @PublicHealthPod on Bluesky @JohnsHopkinsSPH on Instagram @JohnsHopkinsSPH on Facebook @PublicHealthOnCall on YouTube Here's our RSS feed Note: These podcasts are a conversation between the participants, and do not represent the position of Johns Hopkins University.
On tonight's program: The build out of Alligator Alcatraz is on hold. At least for the time being; It seems Texas and California aren't the only states thinking about redrawing congressional district maps; Pill mills used to be a big problem in Florida. Now, it appears that problem may be emerging again; More state money is on its way to battling Sickle Cell Disease in Florida; Despite protests, it looks like some colorful street art that flies in the face of official state policy in Florida may be disappearing; A federal plan to lower the sugar requirement for orange juice may help Florida's struggling citrus industry; A green sea turtle, rescued earlier this year and nursed back to health, is helping researchers find out more about her species; And we'll be among the first to hear the sound of a stingray eating lunch, and learn why these unique sea creatures are perhaps more important than we realized.
So far in 2025, the Trump administration has cut more than $1 billion in NIH grants. That includes a study on sickle cell disease, a blood disorder that affects roughly 100,000 people in the U.S. According to the CDC, 90% of them are Black. Ali Rogin speaks with Dr. Charity Oyedeji, a Duke University hematologist whose research grant was terminated, to learn more. PBS News is supported by - https://www.pbs.org/newshour/about/funders
So far in 2025, the Trump administration has cut more than $1 billion in NIH grants. That includes a study on sickle cell disease, a blood disorder that affects roughly 100,000 people in the U.S. According to the CDC, 90% of them are Black. Ali Rogin speaks with Dr. Charity Oyedeji, a Duke University hematologist whose research grant was terminated, to learn more. PBS News is supported by - https://www.pbs.org/newshour/about/funders
Send us a Text Message (please include your email so we can respond!)Episode 66! Today we talk about Acute Chest Syndrome in Sickle Cell Disease with one of our favorite hematologists, Benjamin Tillman! We base our discussion around the TASC trial or "Comparison of Prophylactic and Therapeutic Doses of Anticoagulation for Acute Chest Syndrome in Sickle Cell Disease" published by Dessap et al in AJRCCM April of 2025.Pubmed: https://pubmed.ncbi.nlm.nih.gov/40209087/AJRCCM: https://www.atsjournals.org/doi/10.1164/rccm.202409-1727OCIf you enjoy the show be sure to like and subscribe, leave that 5 star review! Be sure to follow us on the social @icucast for the associated figures, comments, and other content not available in the audio format! Email us at icuedandtoddcast@gmail.com with any questions or suggestions! Thank you Mike Gannon for the intro and exit music!