Podcasts about attr

On this week's episode, Sam Fazeli, Eric Schmidt, and Brian Skorney are joined by Roivant's Matthew Gline to discuss data, deals and the latest policy updates. The episode kicks off with concerns over potential U.S. Department of Health and Human Services (HHS) funding cuts for domestic HIV programs, with the group analyzing the impact on companies like Gilead and GSK, whose HIV prevention drugs have driven significant investor enthusiasm. They then discuss the uncertainty surrounding the new administration's commitment to public health initiates, considering Robert F. Kennedy Jr.'s appointment as HHS Secretary and his past controversial views on vaccines and HIV prevention. The conversation then shifts to data, including positive results from Immunovant and Roivant's Phase 3 study of batoclimab for myasthenia gravis. The group also examines the surprising absence of short sellers engaging with Roivant post-data release, highlighting shifts in biotech trading strategies. In the muscular dystrophy space, new exon-skipping therapies from Avidity and Dyne show unprecedented dystrophin restoration, suggesting potential functional benefits. However, the tragic death of a patient treated with Sarepta's gene therapy, Elevidys, reignites concerns about accelerated approvals and the FDA's risk-benefit calculus in rare disease research. On the deal-making front, AstraZeneca's $425M acquisition of stealth-mode cell therapy startup EsoBiotec and Sanofi's $600M deal with Dren Bio underscore biotech's valuation disconnect, where early-stage private assets command premium prices while public biotechs struggle. The episode closes with insights into Alnylam's broad label win for Amvuttra in ATTR cardiomyopathy and AstraZeneca's $2.5B investment in biotech agreements and manufacturing in China, reflecting its long-term strategic vision despite ongoing geopolitical tensions. *This episode aired on March 21, 2025

Philip's journey with ATTR began with unexplained weight loss and gastrointestinal issues that puzzled doctors for years. Despite seeing multiple specialists, his condition remained undiagnosed until a physician at Johns Hopkins finally connected the dots, confirming ATTR. By then, Philip had lost 60 pounds and was on the brink of survival. In today's episode of On Rare, David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Senior Director of Patient Advocacy at BridgeBio Gene Therapy, speak with Philip and his wife, Sally, who has helped to navigate the frustrating medical system, advocate for answers, and ultimately helped Philip receive life-saving IV nutrition. After years of uncertainty, Philip finally found an expert team with the knowledge, treatment, and community they needed. Philip and Sally share their story of perseverance, partnership, and hope for a brighter future. Dr. Adam Castaño, head of the Amyloid Prevention Program at BridgeBio, provides a medical overview of TTR amyloidosis (ATTR), a rare, progressive disease caused by misfolded transthyretin (TTR) proteins that form amyloid deposits in different parts of the body such as the heart, the peripheral nervous system, and the gastrointestinal tract. These deposits lead to debilitating symptoms including heart failure, neuropathy, and digestive issues, often mimicking common age-related conditions and possibly contributing to underdiagnosis. ATTR can be hereditary, but it also develops in the general population (this is called wild type). Advancements in non-invasive imaging and specialized protein staining have improved early detection, offering hope for better disease management.

Watch here for a video interview with JACC Associate Editor Michelle Kittleson, MD, FACC, and author Mathew S. Muarer, MD, FACC, as they discuss Dr. Maurer's study published in JACC and presented at ACC.25. This exploratory analysis of HELIOS-B assessed the efficacy of vutrisiran versus placebo in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) by subgroups of baseline heart failure severity (primarily by NYHA class and NT-proBNP levels). Vutrisiran showed evidence of benefit vs placebo on mortality, cardiovascular events, functional capacity, quality of life, and cardiac biomarkers across the range of baseline disease severities in patients enrolled in HELIOS-B, with greatest benefit observed in patients with earlier, less severe disease.

Host: Michelle Kittleson, MD, PhD Transthyretin cardiac amyloidosis (ATTR-CM) is an underrecognized disorder, leading to a variety of unmet needs for patients. Dr. Michelle Kittleson, Director of Postgraduate Education in Heart Failure and Transplantation and Professor of Medicine at the Cedars-Smidt Heart Institute, explores these challenges and discusses how early recognition, genetic screening, and emerging therapies can help improve patient outcomes.

In this episode, Dr. Valentin Fuster highlights a groundbreaking study on acoramides, a novel treatment for transthyretin amyloid cardiomyopathy (ATTR), which shows significant reductions in mortality and cardiovascular hospitalizations compared to a placebo. Experts discuss the clinical implications, comparing acoramides to the previously approved tafamidis, noting exciting advancements in treatments for a once-untreatable disease.

CardioNerds Cardiac Amyloidosis Series Chair Dr. Rick Ferraro and Episode Lead Dr. Anna Radakrishnan discuss the biology of transthyretin amyloid cardiomyopathy (ATTR-CM ) with Dr. Daniel Judge.  Notes were drafted by Dr. Anna Radakrishnan. The audio was engineered by student Dr. Julia Marques.  This episode provides a comprehensive overview of transthyretin (ATTR) cardiac amyloidosis, a complex and rapidly evolving disease process. The discussion covers the key red flags for cardiac amyloidosis, the diagnostic pathway, and the implications of hereditary versus wild-type ATTR. Importantly, the episode delves into the current and emerging therapies for ATTR, including stabilizers, gene silencers, and promising treatments like CRISPR-Cas9 and antibody-based approaches. Dr. Judge shares his insights and excitement about the rapidly advancing field, highlighting the need for early diagnosis and the potential to improve long-term outcomes for patients with this condition.  Enjoy this Circulation Paths to Discovery article to learn more about the CardioNerds mission and journey.  US Cardiology Review is now the official journal of CardioNerds! Submit your manuscripts here.  CardioNerds Cardiac Amyloid PageCardioNerds Episode Page Pearls: - Biology of Transthyretin amyloid cardiomyopathy Maintain a high index of suspicion! Look for subtle (yet telling) signs like ventricular hypertrophy, discordant EKG findings, bilateral carpal tunnel syndrome, and spontaneous biceps tendon rupture.  Utilize the right diagnostic tests. Endomyocardial biopsy remains the gold standard, but non-invasive tools like PYP scan with SPECT imaging and genetic testing are essential for accurate diagnosis.  Differentiating hereditary from wild-type ATTR is critical, as genetic forms may have a more aggressive course and familial implications.  Early diagnosis and intervention significantly improve prognosis, making vigilance in screening and prompt treatment initiation essential.  The future is now! Cutting-edge therapies are transforming the treatment landscape, including TTR stabilizers, gene silencers, and emerging technologies like CRISPR-Cas9 and antibody-based treatments.  Notes - Biology of Transthyretin amyloid cardiomyopathy What is transthyretin amyloid (aTTR) and how is it derived?  Transthyretin (TTR) is a transport protein primarily synthesized by the liver, responsible for carrying thyroid hormones (thyroxine) and retinol (vitamin A) in the blood. It circulates as a tetramer, composed of four identical monomers, which is essential for its stability and function.  In transthyretin amyloid (ATTR) amyloidosis, the TTR protein becomes unstable, leading to its dissociation into monomers. These monomers misfold and aggregate into insoluble amyloid fibrils, which deposit extracellularly in tissues such as the heart, nerves, and gastrointestinal tract. This progressive amyloid deposition leads to organ dysfunction, including restrictive cardiomyopathy and neuropathy.  There are two main forms of ATTR amyloidosis: hereditary (variant) and wild-type (senile) ATTR.  Hereditary ATTR (ATTRv) is caused by mutations in the TTR gene. These mutations destabilize the TTR tetramer, making it more prone to dissociation. This increases misfolding and amyloid fibril formation, resulting in systemic amyloid deposition.   Wild-type ATTR (ATTRwt) occurs without genetic mutations and is primarily age-related. Over time, even normal TTR tetramers can become unstable, leading to gradual misfolding and amyloid deposition, particularly in the heart. ATTRwt is a common but often underdiagnosed cause of heart failure with preserved ejection fraction (HFpEF) in elderly individuals.  How does aTTR lead to deleterious effects in the heart and other organ systems?    Transthyretin amyloidosis leads to organ dysfunction through the deposition of misfolded TTR protein as amyloid fib...

A comprehensive overview of transthyretin amyloidosis, its treatment, barriers to treatment, and future challenges to be aware of with authors, Drs. Erika L. Hellenbart and Robert J. DiDomenico. Full text of the manuscript is available at: https://accpjournals.onlinelibrary.wiley.com/doi/10.1002/phar.4639.

6th AHA 2024: ATTR Cardiomyopathy

Join CardioNerds Heart Failure Section Chair Dr. Jenna Skowronski, episode lead Dr. Apoorva Gangavelli, and expert faculty Dr. Ronald Witteles as they discuss the Nex-Z trial. This was a phase 1, open-label trial investigating nex-z, a CRISPR-Cas9-based treatment, in 36 patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The primary objectives were aimed at studying the safety and pharmacodynamics of this novel gene-based treatment modality. This episode dives into the nuances of the data, future directions for investigation, and future clinical implications. CardioNerds Journal Club PageCardioNerds Episode PageCardioNerds AcademyCardionerds Healy Honor Roll CardioNerds Journal ClubSubscribe to The Heartbeat Newsletter!Check out CardioNerds SWAG!Become a CardioNerds Patron! References - The Nex-Z Trial Fontana, M., Solomon, S. D., Kachadourian, J., Walsh, L., Rocha, R., Lebwohl, D., Smith, D., Täubel, J., Gane, E. J., Pilebro, B., Adams, D., Razvi, Y., Olbertz, J., Haagensen, A., Zhu, P., Xu, Y., Leung, A., Sonderfan, A., Gutstein, D. E., & Gillmore, J. D. (2024). CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. The New England Journal of Medicine. https://doi.org/10.1056/NEJMoa2412309

Στο πρώτο επεισόδιο του 2025, συζητάμε μαζί με τον κ. Αλέξιος Αντωνόπουλος για την καρδιακή αμυλοείδωση. Ποιοι είναι οι κύριοι τύποι καρδιακής αμυλοείδωσης; Πως γίνεται η διάγνωση της καρδιακής αμυλοείδωσης? Σποραδική vs κληρονομική ATTR αμυλοδείδωση. Έχει σημασία; Ποιες είναι οι θεραπείες της αμυλοείδωσης; Που βρισκόμαστε στην Ελλάδα; Καλή ακρόαση!

Featuring articles on advanced melanoma, atrial fibrillation, ATTR amyloidosis, and bladder cancer; a review article on cervical cancer; a Clinical Problem-Solving describing when blurry vision clouds the bigger picture; Medicine and Society's on transitional justice and on rethinking access to HIV medicines; and Perspectives on hepatitis C therapies, on when diversity goals meet multiregional trials, and on miles to go and nowhere to sleep.

Host: John Russell, MD Guest: Noel Dasgupta, MD, FACC Guest: Sami Khella, MD Amyloidosis, often unrecognized, misdiagnosed, and either inappropriately treated or undertreated, is a collection of diseases caused by the misfolding of proteins that aggregate into insoluble amyloid fibrils and deposit in tissues. There are two prominent forms of amyloidosis misfolding of light chain proteins (AL amyloidosis) and transthyretin protein misfolding (ATTR amyloidosis). In this program, two experts in amyloidosis, cardiologist Dr. Noel Dasgupta and neurologist Dr. Sami Khella, will explain the pathophysiology of amyloidosis, describe the prognosis for patients, discuss timely patient screening and diagnostic testing, and provide updated treatment options.

CME credits: 0.50 Valid until: 13-12-2025 Claim your CME credit at https://reachmd.com/programs/cme/the-shifting-attr-cm-landscape-early-diagnosis-emerging-therapies-personalized-care/27090/ Achieving optimal outcomes in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) remains a challenge as diagnosis is often delayed. Patients have a reduced life expectancy and experience debilitating pain and poor quality of life. Hear from the experts as they review the latest information on new and emerging treatment options for transthyretin amyloidosis and how this condition affects patients with cardiomyopathy.=

CME credits: 0.50 Valid until: 13-12-2025 Claim your CME credit at https://reachmd.com/programs/cme/best-practices-for-identifying-diagnosing-and-treating-transthyretin-amyloidosis-attr-pn-and-attr-cm/26799/ Amyloidosis, often unrecognized, misdiagnosed, and either inappropriately treated or undertreated, is a collection of diseases caused by the misfolding of proteins that aggregate into insoluble amyloid fibrils and deposit in tissues. There are two prominent forms of amyloidosis misfolding of light chain proteins (AL amyloidosis) and transthyretin protein misfolding (ATTR amyloidosis). In this program, two experts in amyloidosis, cardiologist Dr. Noel Dasgupta and neurologist Dr. Sami Khella, will explain the pathophysiology of amyloidosis, describe the prognosis for patients, discuss timely patient screening and diagnostic testing, and provide updated treatment options. =

Featuring articles on PCI before transcatheter aortic-valve implantation, treatments in patients with type 2 diabetes, in leprosy, in refractory adult ALL, and in ATTR cardiomyopathy; a review article on drug reaction with eosinophilia and systemic symptoms; a case report of a boy with recurrent fever; and Perspectives on an autopsy of a drug withdrawal, on clearing dense drug-patent thickets, on how a survey change sheds light on iatrogenic opioid use disorder and on being tethered.

Charles, a recently retired cardiologist of 53 years, speaks with David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy, about how his unexpected early diagnosis of ATTR-CM likely impacted the trajectory of his disease. Dr. Jonathan Fox, President and Chief Medical Officer of cardiorenal programs at BridgeBio, explains how ATTR affects the heart and peripheral nervous system, and the importance of early diagnosis.   For years, Charles suffered from numbness and tingling in both wrists and hands. Eventually, when the pain became so severe that operating a mouse for 10 to 15 minutes was difficult, he was referred to a hand surgeon who recommended carpal tunnel surgery. Preparing for the surgery, Charles recalled reading a medical journal article that indicated 10% of people who underwent carpel tunnel surgery had positive tissue biopsy indicative of ATTR-CM. He requested that his surgeon perform a biopsy and shortly thereafter received his diagnosis. The transition from doctor to patient was not easy for Charles. However, he feels fortunate to be a cardiologist, to have read and remembered the article, requested a biopsy, and received the diagnosis more quickly than many others who live with ATTR. “I have three passions: running, traveling, and teaching," Charles shared. The early diagnosis has allowed him to continue to live a full, active life, running nearly daily as he has for the past 45 years. Charles, now 86, continues to serve others, spending his retirement teaching and mentoring health care providers around the country about ATTR and volunteering for amyloidosis advocacy organizations.

For more information regarding this CME/CE activity and to complete the CME/CE requirements and claim credit for this activity, visit:https://www.mycme.com/courses/questions-and-answers-on-amyloid-cardiomyopathy-and-polyneuropathy-9845SummaryIn this activity – which learners can review as a podcast or a webcast - Drs. Chafic Karam and Michelle Kittleson dive into five key questions from recent live-virtual broadcasts about transthyretin amyloidosis (ATTR). They discuss the role of cardiac MRI, biopsy, and genetic testing in diagnosing ATTR cardiomyopathy and polyneuropathy. They also explore treatment options, including silencing therapies and stabilizers, with insights into when patients should be referred for specialized care. The conversation emphasizes the importance of early diagnosis, collaborative care between neurologists and cardiologists, and the expanding therapeutic landscape for ATTR.Learning ObjectivesAt the conclusion of this activity, participants should be better able to:Describe the correct diagnostic algorithm for suspected ATTR, including interpretation of the monoclonal protein screen and indications for biopsy and genetic testing.Initiate appropriate management, including disease-directed therapy and appropriate cardiac care, or referral when indicated.This activity is accredited for CME/CE CreditAssociation of Black Cardiologists, Inc. (ABC) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.Association of Black Cardiologists, Inc. designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.This activity has been planned and implemented in accordance with the Accreditation Standards of the American Association of Nurse Practitioners® (AANP) through the joint providership of the National Association for Continuing Education (NACE) and ABC. NACE is accredited by the AANP as an approved provider of nurse practitioner continuing education. Provider number 121222. This activity is approved for 0.25 contact hours (which does not include hours of pharmacology).In support of improving patient care, this activity has been planned and implemented by Haymarket Medical Education (HME) and NACE. Haymarket Medical Education is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.This knowledge-based activity JA4008232-9999-24-043-H01-P qualifies for 0.25 contact hours (0.025 CEUs) of continuing pharmacy education credits.For additional information about the accreditation of this program, please contact NACE at info@naceonline.com.Summary of Individual DisclosuresPlease review faculty and planner disclosures here.Disclosure of Commercial SupportThis educational activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc., and an educational grant from AstraZeneca Pharmaceuticals.Please visit http://naceonline.com to engage in more live and on demand CME/CE content.

Biopharma had its collective deal-making hat on heading into the Thanksgiving holiday, with Roche buying Poseida for up to $1.5 billion, and Sarepta and Arrowhead tying up in a collaboration potentially worth up to $10 billion. These announcements followed last week's news from Novartis, which snatched up Kate Therapeutics for a little over $1 billion while promising more acquisitions below $5 billion. On the regulatory front, the FDA gave BridgeBio—and transthyretin amyloid cardiomyopathy (ATTR-CM) patients—something to be thankful for late last week with the approval of Attruby for the rare, cardiovascular disease. The nod sets up a potential three-way race with Pfizer's tafamidis and Alnylam's Amvuttra, the latter of which was accepted for FDA review in ATTR-CM on Monday.   On the opposite end of the clinical development spectrum, Cassava Sciences' controversial Alzheimer's drug failed to reduce cognitive or functional decline in a Phase III trial. And investors were unimpressed by the 20% weight loss generated by Amgen's MariTide in a much-anticipated Phase II trial, as the company's stock tumbled 11% Tuesday. Drawing much attention from the biopharma industry, President-elect Donald Trump continued with his nominees for top healthcare posts, announcing COVID-19 critic Marty Makary to lead the FDA and well-known vaccine skeptic Dave Weldon for CDC director. Meanwhile, the FDA is grappling with the loss of Chevron Deference pertaining to a legal challenge over Eli Lilly's GLP-1 shortages. Finally, the next generation of antibody-drug conjugates remains hot, with Danish biotech Adcendo reeling in $135M in a Series B financing round.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Next-generation antibody-drug conjugates (ADCs) are driving a renaissance in the oncology drug class, with companies like ADC Therapeutics, Sutro Biopharma, and Zai Lab developing new ADCs to address challenges with payload and toxicity. BridgeBio recently secured FDA approval for its drug to treat ATTR-CM, launching competition with Pfizer in the market. Meanwhile, Sage Therapeutics faced setbacks with the discontinuation of its lead candidate for Huntington's disease after a third clinical failure. Applied Therapeutics is awaiting FDA verdict on a rare disease therapy, while AstraZeneca successfully defended its drug Andexxa against safety concerns. Other developments include patient death in a Rett syndrome trial, Merck's asthma drug Singulair potentially linked to mental health problems, and Intellia's CRISPR gene editor showing promise in ATTR amyloidosis.The biopharma industry is seeing advancements and challenges across various therapeutic areas, prompting discussions on future coverage topics in neuroscience, oncology, cell & gene therapy, and more.

JACC: Associate Editor Michelle M. Kittleson, MD, PhD, FACC, talks with authors Scott Solomon, MD, FACC and. Marianna Fontana, MD, about their study published in JACC and presented at AHA. Outpatient worsening heart failure (HF) (oral diuretic intensification or initiation) is simple to assess and has been shown to be prognostic of mortality in patients with ATTR-CM. In this pre-specified analysis of a contemporary ATTR-CM population, patients with outpatient worsening HF had an increased risk of all-cause mortality and CV events and all-cause mortality, as well as greater deterioration in assessments of functional capacity, health status, and quality of life. Vutrisiran significantly reduced the risk of outpatient worsening HF and the composite of outpatient worsening HF, all-cause mortality, and recurrent CV events compared with placebo.

Did you miss AHA 2024? Listen here to brief discussions of the latest research. Eric Rubin is the Editor-in-Chief of the Journal. Jane Leopold is a Deputy Editor of the Journal. Stephen Morrissey, the interviewer, is the Executive Managing Editor of the Journal. E.J. Rubin, J. Leopold, and S. Morrissey. NEJM at AHA — CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. N Engl J Med. DOI: 10.1056/NEJMe2414473.

For more information regarding this CME/CE activity and to complete the CME/CE requirements and claim credit for this activity, visit:https://www.mycme.com/courses/management-of-amyloid-cardiomyopathy-and-polyneuropathy-9813SummaryIn this CME/CE podcast episode, Cardiologist Dr. Michelle Kittleson and Neurologist Dr. Chafic Karam discuss the multidisciplinary management of transthyretin amyloidosis (ATTR). They explore how ATTR affects both the heart and the nervous system, focusing on recognizing symptoms, diagnosing the disease, and managing treatment. Dr. Karam shares insights on peripheral neuropathy caused by amyloidosis, including clues that suggest amyloidosis over more common neuropathies. Dr. Kittleson highlights how cardiologists assess and manage TTR cardiomyopathy, emphasizing the importance of early diagnosis and the evolving therapeutic landscape. Together, they discuss the collaborative nature of care, the impact of new therapies on clinical outcomes, and the importance of raising awareness for ATTR.Learning ObjectivesDescribe the correct diagnostic algorithm for suspected ATTR, including interpretation of the monoclonal protein screen and indications for biopsy and genetic testingInitiate appropriate management, including disease-directed therapy and appropriate cardiac care, or referral when indicatedThis activity is accredited for CME/CE CreditAssociation of Black Cardiologists, Inc. (ABC) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.Association of Black Cardiologists, Inc. designates this enduring material for a maximum of 0.25 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.This activity has been planned and implemented in accordance with the Accreditation Standards of the American Association of Nurse Practitioners® (AANP) through the joint providership of the National Association for Continuing Education (NACE) and ABC. NACE is accredited by the AANP as an approved provider of nurse practitioner continuing education. Provider number 121222. This activity is approved for 0.25 contact hours (which does not include hours of pharmacology).For additional information about the accreditation of this program, please contact NACE at info@naceonline.com.Summary of Individual DisclosuresPlease review faculty and planner disclosures here.Disclosure of Commercial SupportThis educational activity is supported by an educational grant from Alnylam Pharmaceuticals Inc., and an educational grant from AstraZeneca Pharmaceuticals.Please visit http://naceonline.com to engage in more live and on demand CME/CE content.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/TDS865. CME credit will be available until October 23, 2025.TTRansforming the TTReatment of ATTR: A Guide to the Pathophysiology, Diagnosis, and Emerging Treatment Strategies for ATTR Amyloidosis In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by an independent educational grant from AstraZeneca LP.Disclosure information is available at the beginning of the video presentation.

JACC: Heart Failure Social Media Editor Giorgia Benzoni, MD, discusses a recently published Leading Edge Commentary comparing the pathophysiological differences between ATTR and AL cardiac amyloidosis and implications for therapy.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Medtronic's chief medical officer of acute care and monitoring has departed for a new role. The FDA has finalized voluntary malfunction summary reporting guidance. Penumbra is laying off 71 people after axing its virtual reality division. Qiagen and AstraZeneca have expanded their companion diagnostic pact. The Association for Molecular Pathology is suing to block an FDA lab test rule. Layoffs in the medical device industry continue to be a trend. Industry news includes the launch of the first over-the-counter glucose monitor, unexpected partnerships in diabetes tech, and pushback on FDA regulation of lab-developed tests.The text discusses various updates in the biopharma industry, including Alnylam's heart drug data sparking debate, Novo building a heart failure case for semaglutide, and a slump in cell and gene therapy investment. The industry is shifting towards more patient-centric commercialization strategies, with companies like Pfizer and Lilly breaking into the direct-to-consumer market. The summer has been a mixed bag for biotech, with market fluctuations and limited IPOs and acquisitions. Alnylam's full study data on its heart drug vutrisiran showed benefits but also raised skepticism. Novo's analysis showed semaglutide's effectiveness in reducing heart failure risk. Additionally, PBM executives are facing fines for alleged perjury in a house hearing defending their business practices.The text discusses the pricing and sales of gene therapies, particularly focusing on Novartis' Zolgensma, which has been the only gene therapy to cross the blockbuster threshold with sales of $1.2 billion in 2021 and $1.4 billion in 2022. While high price tags have not always resulted in significant returns for pharmaceutical companies with gene therapies, Zolgensma has been an exception. Other companies have faced challenges with new gene therapies, such as Biomarin Pharmaceuticals, which recently announced layoffs due to dismal sales of its hemophilia A gene therapy Roctavian.Alnylam Pharmaceuticals has released detailed data on their drug Vutrisiran, showing its benefits in treating a progressive and fatal heart condition known as ATTR cardiomyopathy. The results were published in the New England Journal of Medicine. While the data confirms the therapy's benefit, there are still questions about how exactly Vutrisiran should be used in treating the disease. The study is expected to continue to fuel debate among doctors.The text discusses the recent slump in investment in cell and gene therapy, with fewer venture funding rounds closed by developers in the first six months of 2024. Two companies, Biomarin and Tome Biosciences, are cutting jobs, with over 200 and over 100 layoffs respectively. Biomarin has made changes to its executive team and drug pipeline to refocus resources. Additionally, Bayer has partnered with RNA drugmaker Nextrna Therapeutics to develop new cancer therapies. The text also highlights the challenges faced in clinical trials, with nearly 80% failing to meet enrollment goals and schedules. The importance of understanding social determinants of health for research is emphasized.Biopharma Dive's Gene Therapy Weekly provides news and insights on gene therapy for biopharma leaders.

Isabel Conceição, MD - Prioritizing Patient-Centered Care for ATTR: Expert Perspectives on the Evolution of Data and Decision-Making

Isabel Conceição, MD - Prioritizing Patient-Centered Care for ATTR: Expert Perspectives on the Evolution of Data and Decision-Making

Sean joins David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy to share his journey with ATTR. In this episode, Sean describes his initial symptoms, which included carpal tunnel syndrome and numbness in his foot and ankle, the misdiagnosis he experienced, and his eventual diagnosis at the Cleveland Clinic in Abu Dhabi. While the diagnosis was a relief it was also difficult to accept and took an emotional toll on him and those around him. Sean has found this to be a common experience of many diagnosed with ATTR. Since Sean's form of ATTR is hereditary, various members of his family have also been tested. Sean also highlights the positive changes in his life, including meeting his wife, Robin, discovering a talent for drawing, and becoming a public speaker about ATTR. Jonathan Fox, President and Chief Medical Officer of cardiorenal programs at BridgeBio, joins us again to give a medical introduction to ATTR. On this episode, Jonathan highlights the differences of the ATTR T80 variant. Previously, we learned that ATTR amyloidosis is caused by the dissociation of a protein called transthyretin, or TTR, that changes its shape and forms into fibrous clumps. These clumps of misshapen protein are deposited into various organs and peripheral nerves, including the heart, which can cause them to function abnormally. Jonathan explains the possible origin of the T80 variant and how the T80 variant symptoms can differ from ATTR-CM.   To learn more about living with amyloidosis visit Mackenzie's Mission, www.mm713.org

On this week's Biotech Hangout, hosts Daphne Zohar, Brad Loncar, Josh Schimmer, Tim Opler and Chris Garabedian open up with a conversation on recent trends in M&A for private biotechs versus public companies and the potential impact of the election year. The hosts also cover the important data readouts from the week, including Alnylam's positive Phase 3 results for its ATTR drug and Lyell's report of a patient death in its early-stage CAR T trial. Additional data covered includes Wave's Huntington data readout, Intellia's redosing of CRISPR gene editing therapy and Savara's Phase 3 readout in rare lung disease. In financing news, the hosts recap Curie.bio's $380M Pro Rata Series A Fund, Formation's $372M Series D and Recursion's $200M offering. This leads into a conversation on AI in drug development and how this is an area full of both promise and hype. On the management theme, the group discusses Gingko's recent layoffs, business model & revenues. Rounding out the show on the management theme, Amylyx's pivot to license GLP-1 was mentioned as an example of a management team earning credibility for following through on their word. *This episode aired on June 28, 2024.

JACC: CardioOncology Deputy Editor Ronald Witteles, MD and Mathew Maurer, MD discuss the present and future state of ATTR amyloidosis diagnosis and treatment.

JACC: CardioOncology Deputy Editor Ronald Witteles, MD and Mazen Hanna, MD discuss some of the latest data from the 2024 International Society of Amyloidosis meeting.

CME credits: 0.25 Valid until: 22-05-2025 Claim your CME credit at https://reachmd.com/programs/cme/strategic-excellence-mastering-the-expanding-attr-cm-therapeutic-landscape-in-pharmacy-practice/18124/ Join us in this educational endeavor to empower pharmacists with the latest insights and strategies for improved decision-making in ATTR-CM management. The program emphasizes the latest clinical insights in this evolving therapeutic landscape, addressing changes in diagnostic approaches over the last decade, and providing a nuanced perspective on ATTR-CM progression for more effective patient care. Don't miss out on this opportunity to elevate the standard of care for your patients.=

CME credits: 0.25 Valid until: 09-05-2025 Claim your CME credit at https://reachmd.com/programs/cme/attr-cm-management-pearls-from-recent-clinical-trials-and-tailoring-therapy/18005/ Struggling to manage ATTR-CM? Our faculty are exploring the key considerations shaping current clinical trial design and how innovations might change our approach to the management of ATTR-CM in the future. They'll also discuss the use of outpatient diuretic intensification as a potential endpoint in trials and the ongoing challenge of defining and measuring “clinically meaningful benefit” for patients with ATTR-CM. Don't miss this opportunity to gain valuable insights and elevate your understanding of ATTR-CM treatment strategies!=

In part two, Eric continues talking about his experience living with Transthyretin Amyloidosis (ATTR) with David Rintell, Head of Patient Advocacy at BridgeBio, and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapy. Eric recalls the surprise he experienced when a routine doctor's visit led to him, within hours, to undergoing a heart transplant. Though the surgery was successful, Eric describes his complex recovery from the transplant. Age, old sports injuries, and lingering ATTR symptoms all contributed to a year-long recovery process, which included a two-week stay in the hospital, followed by an acute rehabilitation facility, a few weeks in a hotel, and several more months of recovery at home. Eric ends the conversation by stressing the importance of community – strong connections with other people were not only crucial to his recovery, but also opened doors for mentorship, activism, and his current involvement with the Northern California Amyloidosis Support Group.

CME credits: 1.00 Valid until: 23-04-2025 Claim your CME credit at https://reachmd.com/programs/cme/attr-amyloidosis-familial-vs-wild-type/24132/ Deepen your understanding of hereditary transthyretin amyloid polyneuropathy (ATTRv-PN) with this comprehensive CME series! Gain valuable insights from patient cases, expert lectures, and interviews. Explore the complexities of ATTRv-PN, differentiate familial vs wild-type forms, identify red flags for early detection, and review the latest diagnostic tools. Discover promising new therapies and what the future may hold for ATTRv-PN treatment. Join the experts and equip yourself to effectively manage this challenging disease and improve patient outcomes.

Commentary by Bonnie Ky

Eric joins David Rintell, Head of Patient Advocacy at BridgeBio and Mandy Rohrig, Director of Patient Advocacy at BridgeBio Gene Therapyin a two-part conversation to talk about his experience of living with Transthyretin Amyloidosis (ATTR). Eric was an extremely active person who enjoyed sports, riding horses and working on his 20-acre property when he began to have issues which he now attributes to ATTR. Eric's diagnostic journey took 14 years and as the disease progressed, he experienced two carpal tunnel surgeries, and several other cardiac procedures. In fact, by the time he was diagnosed, Eric could not walk the length of his home (60 feet) without getting out of breath. Four years after his diagnosis he received a heart transplant. Despite the difficulties, Eric highlights the positives of this diagnosis and how living with ATTR and engaging with the ATTR community has taught him to be intellectually curious, empathetic, grateful for the care he has received and happy to be alive.  Jonathan Fox, President and Chief Medical Officer of cardiorenal programs at BridgeBio. Jonathan explains the causes of ATTR and what happens to people living with it. ATTR amyloidosis is caused by the dissociation of a protein called transthyretin, or TTR, that changes its shape and forms into fibrous clumps. These clumps of misshapen protein are deposited into various organs and peripheral nerves, including the heart, which can cause them to function abnormally.

Commentary by Dr. Valentin Fuster

Genealogist and health educator Bernice Bennett, host of Alnylam's  Family Health History Road Trip program, traveled and visited families affected by an inherited condition called hereditary ATTR amyloidosis – She is joined by Giselle, whose father's diagnosis solved her own health puzzle and why it's so important for families to discuss their health history with each other, and with their doctors. 

Genealogist and health educator Bernice Bennett, host of Alnylam's  Family Health History Road Trip program, traveled and visited families affected by an inherited condition called hereditary ATTR amyloidosis – She is joined by Giselle, whose father's diagnosis solved her own health puzzle and why it's so important for families to discuss their health history with each other, and with their doctors. 

Systemic amyloidosis from transthyretin (ATTR) protein is increasingly recognized as an important cause of heart failure in older people. JAMA Deputy Editor Mary McGrae McDermott, MD, discusses the diagnosis and treatment of ATTR protein cardiomyopathy with Frederick L. Ruberg, MD, of the Boston University Chobanian & Avedisian School of Medicine. Related Content: Cardiac Amyloidosis Due to Transthyretin Protein

ESC TV Today brings you concise analysis from the world's leading experts, so you can stay on top of what's happening in your field quickly. This episode covers: Cardiology This Week: A concise summary of recent studies Management of cardiac amyloidosis e-cigarettes and cardiovascular disease Statistics Made Easy: The Hazard Ratio Host: Susanna Price Guests: Carlos Aguiar, Julian Gillmore, Maryam Kavousi Want to watch that episode? Go to: https://esc365.escardio.org/event/1144   Disclaimer  This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC.   Declarations of interests Stephan Achenbach, Maryam Kavousi, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, Lilly, Novartis, Pfizer, Sanofi, Servier, Tecnimede. Davide Capodanno has declared to have potential conflicts of interest to report: Sanofi, Novo Nordisk, Terumo, Medtronic. Julian Gillmore has declared to have potential conflicts of interest to report: consultancy for Alnylam, ATTRalus, AstraZeneca, Bridgebio, Intellia, Ionis, Lycia. Emma Svennberg has declared to have potential conflicts of interest to report: institutional research grants from Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Boehringer-Ingelheim, Johnson & Johnson, Merck Sharp & Dohme.

Visit nascentmc.com/podcast for full show notes [free course] ChatGPT4 in medical writing and editing at learnAMAstyle.com Nascentmc.com for medical writing assistance for your CME or Medical Communications company. Eplontersen for ATTR-CM Eplontersen received FDA Fast Track designation for treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults, aiming to inhibit TTR protein production. Nivolumab for NSCLC The FDA accepted supplemental applications for nivolumab in resectable stage 2A to 3B non-small cell lung cancer (NSCLC) based on the CheckMate-77T trial showing improved survival rates. RSV Vaccine for Adults Aged 50 to 59 The FDA prioritizes review of GSK's Arexvy vaccine for RSV in adults aged 50-59 at risk of complications, expanding from its existing approval for those 60 and older. Spinal Cord Stimulation System The FDA approved Boston Scientific's WaveWriter Spinal Cord Stimulation Systems for chronic low back and leg pain treatment in non-surgery patients, based on the SOLIS trial results. OK-101 for Neuropathic Corneal Pain The FDA approved an IND application for OK-101, a first for treating neuropathic corneal pain (NCP), an Orphan disease, developed by OKYO Pharma Limited. Viz ICH Plus for Brain Bleed The FDA cleared Viz ICH Plus, an AI algorithm by Viz.ai for automating the identification and quantification of brain bleeds and structures in NCCT images. Vepdegestrant for MBC Vepdegestrant received FDA Fast Track designation for treating ER-positive/HER2-negative advanced or metastatic breast cancer in patients previously treated with endocrine therapy. VerTouch Spinal Puncture Device The FDA cleared VerTouch, a handheld imaging tool by IntuiTap Medical, designed to improve the accuracy of spinal punctures by providing a 2D image of lumbar spinal anatomy.

The following question refers to Section 7.8 of the 2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure.The question is asked by Stony Brook University Hospital medicine resident and CardioNerds Intern Dr. Chelsea Tweneboah, answered first by Mayo Clinic Cardiology Fellow and CardioNerds Academy Chief Dr. Teodora Donisan, and then by expert faculty Dr. Michelle Kittleson.The Decipher the Guidelines: 2022 AHA / ACC / HFSA Guideline for The Management of Heart Failure series was developed by the CardioNerds and created in collaboration with the American Heart Association and the Heart Failure Society of America. It was created by 30 trainees spanning college through advanced fellowship under the leadership of CardioNerds Cofounders Dr. Amit Goyal and Dr. Dan Ambinder, with mentorship from Dr. Anu Lala, Dr. Robert Mentz, and Dr. Nancy Sweitzer. We thank Dr. Judy Bezanson and Dr. Elliott Antman for tremendous guidance.Enjoy this Circulation 2022 Paths to Discovery article to learn about the CardioNerds story, mission, and values. Question #29 A 69-year-old man was referred to the cardiology clinic after being found to have a reduced left ventricular ejection fraction and left ventricular hypertrophy. For the last several months he has been experiencing progressively worsening fatigue and shortness of breath while getting to the 2nd floor in his house. He has a history of bilateral carpal tunnel syndrome and chronic low back pain. He takes no medications. On exam, his heart rate is 82 bpm, blood pressure is 86/60 mmHg, O2 saturation is 97% breathing ambient air, and BMI is 29 kg/m2. He has a regular rate and rhythm with normal S1 and S2, bibasilar pulmonary rales, and 1+ pitting edema in both legs. EKG shows normal sinus rhythm with a first-degree AV delay and low voltages. Transthoracic echocardiogram shows a moderately depressed LVEF of 35-39%, severe concentric hypertrophy with a left ventricular posterior wall thickness of 1.5 cm and strain imaging showing globally reduced longitudinal strain with apical sparring. There is also biatrial enlargement and a small pericardial effusion. A pharmacologic nuclear stress test did not reveal any perfusion defects. A gammopathy panel including SPEP, UPEP, serum and urine immunofixation studies, and serum free light chains are unrevealing. A 99mTc-Pyrophosphate scan was positive with grade 3 uptake. In addition to starting diuretics, what is the next most appropriate step for managing for this patient? A Start metoprolol succinate B Start sacubitril/valsartan C Perform genetic sequencing of the TTR gene D Perform endomyocardial biopsy Answer #29 Explanation The correct answer is C – perform genetic sequencing of the TTR gene.   This patient has findings which raise suspicion for cardiac amyloidosis. There are both cardiac (low voltages on EKG and echocardiogram showing marked LVH with biatrial enlargement and small pericardial effusion as well as a characteristic strain pattern) and extra-cardiac (bilateral carpal tunnel syndrome and low back pain) features to suggest amyloidosis. The diagnosis of cardiac amyloidosis requires a high index of suspicion and most commonly occurs due to a deposition of monoclonal immunoglobulin light chains (AL-CM) or transthyretin (ATTR-CM). ATTR may cause cardiac amyloidosis as either a pathogenic variant (ATTRv) or as a wild-type protein (ATTRwt).   Patients for whom there is a clinical suspicion for cardiac amyloidosis should have screening for serum and urine monoclonal light chains with serum and urine immunofixation electrophoresis and serum free light chains (Class 1, LOE B-NR). Immunofixation electrophoresis (IFE) is preferred because serum or urine plasma electrophoresis (SPEP or UPEP) are less sensitive. Together, measurement of serum IFE, urine IFE, and serum FLC is >99% sensitive for AL amyloidosis.