Scholar Rock Holding has resubmitted its application for its spinal muscular atrophy treatment to the Food and Drug Administration. The biopharmaceutical company said that it has sent in a new ...
A first-in-human clinical trial evaluating an investigational gene therapy targeting complement-mediated pathways in ...
Biogen Inc. BIIB shares are up on Monday following the FDA’s approval of a new high-dose regimen for Spinraza (nusinersen), a ...
Full 12-month data from Ocugen’s phase 2 eye disease gene therapy show the therapy significantly reduces the size of lesions, ...
As adherence to the Mediterranean-DASH Intervention for Neurodegenerative Delay diet went up, total grey matter decline and ...
This high dose nusinersen regimen for spinal muscular atrophy includes 50mg loading doses and 28mg maintenance doses.
Spinal and bulbar muscular atrophy (SBMA) is a rare inherited disease that causes progressive muscle weakness and wasting in ...
Geographic atrophy (GA) is a chronic, progressive degeneration of the macula that can occur in some people with dry macular degeneration. GA is a late, or advanced, stage of the disease. The people ...
Mission MSA is proud to announce its 2026 designated MSA Centers of Excellence (COE), a prestigious network of clinical institutions ...
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