Option Care Health Inc., the largest U.S. independent provider of home and alternate site infusion services, was selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy who are amenable to exon 53 skipping therapy.
A roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for Covid-19, the illness caused by the virus.
Argenx announced positive topline results from the company’s Phase III ADAPT clinical trial of efgartigimod for myasthenia gravis.
Biogen and Sangamo Therapeutics announced a broad global licensing collaboration deal to develop and commercialize several compounds for a range of neurological and neuromuscular diseases.
Roche Holding AG made the company’s second major gene therapy deal in a year, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc.’s investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States.
Reata Pharmaceuticals Inc.’s Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met the primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale relative to placebo after 48 weeks of treatment.
Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.
The U.S. FDA gave Alexion Pharmaceuticals a thumbs-up for Soliris (eculizumab) to treat neuromyelitis optica spectrum disorder in adults that express a specific biomarker.
The return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
Shares of Voyager Therapeutics skyrocketed after the company announced a collaborative deal to develop a gene therapy treatment for Parkinson’s disease with Neurocrine Biosciences.