News FDA gives the go-ahead to restart Elevidys shipments Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.
R&D Novel study designs for rare diseases Developing treatments for rare diseases poses many unique challenges that cannot be addressed by traditional study designs.
News Sarepta backs down and pauses Elevidys shipments The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.
News FDA names a new CDER director, choosing a biotech vet The FDA has rounded out its senior leadership by naming biopharma industry executive George Tidmarsh to the role of CDER director.
News Sarepta resists as FDA seeks pause on Elevidys use Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.
News GSK's growth plans dented by Blenrep setback FDA advisors have voted that the benefit/risk profile of GSK's multiple myeloma therapy, Blenrep, does not support its approval in the US.
News Lilly's 'triple G' retatrutide aces crucial obesity trial Lilly's retatrutide achieved weight loss of more than 30% at two years in a new trial, firming up its credentials as a next-generation obesity drug.
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