Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, unveiled positive results in its sevasemten programme for Becker and Duchenne muscular dystrophies. The company announced positive data from MESA, an open label extension …
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy …
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, the agency is investigating two Duchenne muscular dystrophy patient deaths following treatment with Sarepta Therapeutics’ Elevidys. …
REGULATION FDA probes deaths of boys taking Sarepta Duchenne drug The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne muscular dystrophy who were treated with Sarepta Therapeutics’ gene …
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A panelist discusses how a 16-year-old DMD patient shows more rapid disease progression despite early steroid treatment, with early onset cardiac issues, scoliosis, and increasing upper limb weakness requiring comprehensive specialist care.
A panelist discusses how older DMD patients might benefit from newer genetic therapies including gene therapy and exon skipping, though eligibility depends on specific mutations and absence of pre-existing immunity to AAV vectors.
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