BioCentury This Week
BioCentury This Week
BioCentury
Ep. 239 - Rare Disease Spotlight: MPS & DMD
24 minutes Posted Jun 25, 2024 at 12:00 am.
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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 ap...