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	Podcast:		BioCentury This Week
	Episode:		Ep. 239 - Rare Disease Spotlight: MPS & DMD
	Category:		Science & Medicine
	Duration:		00:24:59
	Publish Date:		2024-06-25 00:00:00
	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 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/652789 00:01 - Sponsor Message: Nxera Pharma 02:05 - Introducing BioCentury's Grand Rounds 06:46 - An MPS Breakthrough 14:12 - Sarepta Approval
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