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Podcast: BioCentury This Week
Episode: Ep. 239 - Rare Disease Spotlight: MPS & DMD
Description: 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 geneti...