Still, investigators say there are gaps in spinal muscular atrophy (SMA) screening protocol that need to be filled. New therapies have helped to improve outcomes for patients with spinal muscular ...
Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad group of patients aged 2 to ...
SOUTH PLAINFIELD, N.J., Jan. 22, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), the SMA Foundation, and Roche (SIX: RO, ROG; OTCQX: RHHBY), announced ...
First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi ® (risdiplam) ...
CAMBRIDGE, Mass. and SAN FRANCISCO, Aug. 20, 2019 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Invitae Corporation (NYSE: NVTA) today announced that SMA STAT, a new, rapid-turnaround genetic ...
Testing for spinal muscular atrophy in an Australian newborn screening program identified patients across socioeconomic and cultural demographics, mitigating inequity and providing patients with ...
In August, Repligen (RGEN 3.76%) decided to get out of the drug discovery business and focus on manufacturing biologic drugs, so it wasn't a big surprise today when the company announced that it had ...
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