Ep. 239 - Rare Disease Spotlight: MPS & DMD

A biomarker breakthrough could shave years off the development of therapies for a group of devastating, progressive ultrarare diseases. On the latest BioCentury This Week podcast, BioCentury’s editors discuss FDA’s agreement to allow biopharma companies to use a biomarker as the basis for accelerated approval of neuronal mucopolysaccharidoses therapies, which marks an important inflection point, both for MPS and rare diseases in general. The editors also assess FDA’s decision to grant full approval to and expand the label of Sarepta DMD gene therapy Elevidys delandistrogene moxeparvovec, igniting another controversy over the agency's decision-making; discuss how human genetics is promising to remove some of the risk from neurology drug development; and preview BioCentury’s new conference, Grand Rounds, which takes place in September in Nashville, at the interface of academia and industry. This week’s podcast is sponsored by Nxera Pharma.View full story: https://www.biocentury.com/article/65278900:01 - Sponsor Message: Nxera Pharma02:05 - Introducing BioCentury's Grand Rounds06:46 - An MPS Breakthrough14:12 - Sarepta ApprovalReach us by sending a text