Pfizer Inc. said on April 28 the company would open the first U.S. trial sites for its experimental gene therapy for a muscle-wasting disorder, after the Food and Drug Administration lifted the regulatory agency’s hold on a late-stage study.
Solid Biosciences Inc. announced organizational structure changes to create a leaner company focused on advancing SGT-001, a gene transfer candidate for the treatment of Duchenne muscular dystrophy.
Two companies that specialize in AI-backed drug development – Insilico Medicine and A2A Pharmaceuticals – are teaming up to take on the rare disease known as Duchene Muscular Dystrophy.
Sarepta Therapeutics shares jumped 60 percent after reporting promising results from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD).
Swiss drugmaker Santhera’s shares plunged nearly 60 percent after a European panel recommended against approving one of the company’s drugs to be used in patients with Duchenne muscular dystrophy (DMD).
Charles Seife, a journalist, author and professor at New York University, filed a federal lawsuit against the U.S. Food and Drug Administration related to documents over the approval of Sarepta Therapeutics’ Exondys 51 for Duchenne muscular dystrophy (DMD).
PTC Therapeutics Inc. said it would stop developing its cystic fibrosis drug after it failed to meet the main goal of a late-stage study.
Marathon Pharmaceuticals is “pausing” the launch of its Duchenne muscular dystrophy drug after U.S. lawmakers questioned why the company priced it at $89,000 a year.
Pfizer acquired privately held gene therapy developer Bamboo Therapeutics in a deal worth up to $645 million to boost its presence in the treatment of rare diseases.