Ep. 179 - DMD Gene Therapies, CBER & NIH

If FDA approves Sarepta's gene therapy for Duchenne muscular dystrophy, patients will soon face the difficult choice between taking this treatment or waiting for a better option among the “unprecedented” amount of innovation ahead in DMD, as taking both may not be an option, BioCentury Executive Director Lauren Martz said on the latest BioCentury This Week podcast. Martz and colleagues assess the landscape for DMD gene therapies ahead of the May 29 PDUFA date for delandistrogene moxeparvovec.
Also on the show, BioCentury Washington Editor Steve Usdin discusses takeaways from his conversation with FDA's Peter Marks; three tasks for Monica Bertagnolli to prioritize at NIH; and what the debt ceiling means for the life sciences. And on the heels of Bio€quity Europe, Editor in Chief Simone Fishburn delivers her insights from BioCentury’s European conference.
This week’s podcast is sponsored by Cancer Research Horizons.