Gene Therapy Breakthroughs in Genetic Cardiomyopathies
Heart Failure Beat19 Heinä 2025

Gene Therapy Breakthroughs in Genetic Cardiomyopathies

In this episode of the Heart Failure Beat, Dr. Eric Adler of UC San Diego Health joins the show to discuss his landmark gene therapy trial for Danon disease, recently published in the New England Journal of Medicine. Dr. Adler recounts the powerful bedside-to-bench-to-bedside story that led to the development of a groundbreaking treatment for this severe, genetic cardiomyopathy. He explains the mechanics and evolution of gene therapy, its promise in treating monogenic heart failure syndromes, and the early but encouraging clinical outcomes—like dramatic reductions in troponins and heart mass. The episode also explores regulatory and safety challenges, delivery limitations, and the critical role of patient advocacy. Whether you’re a heart failure specialist or a translational researcher, this conversation offers compelling insights into the future of precision therapies in cardiology.

Tämä jakso on lisätty Podme-palveluun avoimen RSS-syötteen kautta eikä se ole Podmen omaa tuotantoa. Siksi jakso saattaa sisältää mainontaa.

Jaksot(51)

A New Dawn in Inflammatory & Infiltrative Cardiomyopathies

A New Dawn in Inflammatory & Infiltrative Cardiomyopathies

The May (2026) issue of the Journal of Cardiac Failure shines a spotlight on infiltrative and inflammatory cardiomyopathies, including amyloidosis, sarcoidosis, and myocarditis, and the rapidly evolvi...

21 Touko 32min

MRAs in HFpEF & HFmrEF: A Clinical Debate

MRAs in HFpEF & HFmrEF: A Clinical Debate

Are mineralocorticoid receptor antagonists (MRAs) ready for prime time in HFpEF and HFmrEF?  In this special debate-format episode of the Heart Failure Beat, two leading heart failure pharmacists, Sto...

26 Maalis 35min

Integrating Technology for Smarter Heart Failure Care

Integrating Technology for Smarter Heart Failure Care

Heart failure care is entering a new era - one defined not by singular devices, but by integrated systems of technology working together. In this episode, Laura Peters, DNP, joins The Heart Failure Be...

19 Helmi 28min

Rethinking Sodium Restriction in Heart Failure: What the Evidence (Doesn’t) Show

Rethinking Sodium Restriction in Heart Failure: What the Evidence (Doesn’t) Show

For decades, sodium restriction has been treated as a cornerstone of heart failure self-care—despite limited trial-quality evidence. In this episode of Heart Failure Beat, Dr. Tariq Ahmad (Yale School...

22 Tammi 25min

HFSA Presidential Voices: Leading the Modern Era of Heart Failure

HFSA Presidential Voices: Leading the Modern Era of Heart Failure

Priya Umapathi, MD speaks with six HFSA past presidents live from ASM 2025. Nancy M. Albert, PhD, CCNS, CCRN, NE-BC, Biykem Bozkurt, MD, PhD, Jim Fang, MD, FHFSA, Mark H. Drazner, MD MSc, Michael Felk...

18 Joulu 202525min

Revisiting the Fundamentals: Hemodynamics at the Heart of Heart Failure Care

Revisiting the Fundamentals: Hemodynamics at the Heart of Heart Failure Care

Guest editors Drs. Mak, Burkhoff, and Belkin join Heart Failure Beat to unpack the Journal of Cardiac Failure Hemodynamics Focus Issue. They discuss how renewed attention to pressure-volume loops, ECM...

15 Marras 202526min

Behind the Pages of the New Journal, JCF-Intersections

Behind the Pages of the New Journal, JCF-Intersections

Meet the editorial team behind JCF-Intersections, HFSA’s and the JCF Family of Journal’s new open-access journal. Drs. Jason Katz and Nosheen Reza share their vision for a journal that sits at the cro...

20 Syys 202528min

Suosittua kategoriassa Tiede

rss-mita-tulisi-tietaa
tiedekulma-podcast
rss-poliisin-mieli
utelias-mieli
rss-tiedetta-vai-tarinaa
university-of-eastern-finland
docemilia
sotataidon-ytimessa
filocast-filosofian-perusteet
menologeja-tutkimusmatka-vaihdevuosiin
rss-bios-podcast
rss-ranskaa-raakana
rss-duodecim-lehti
rss-duokkari-ekstra
rss-astetta-parempi-elama-podcast
rss-ilmasto-kriisissa
rss-ylistys-elaimille
rss-sosiopodi
rss-totuuden-liepeilla