From February 12, all newborn babies will be offered screening for spinal muscular atrophy (SMA), a rare progressive neuromuscular disease. SMA will be one of more than 20 other rare disorders ...
From February 12, all newborn babies will be offered screening for spinal muscular atrophy (SMA), a rare progressive neuromuscular disease. SMA will be one of more than 20 other rare disorders ...
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
In surveys, parents of children with SMA and healthcare professionals in Sweden note the many ways the pandemic affected ...
Parents of children with Spinal Muscular Atrophy (SMA), a rare disease, say the budget is mere eyewash. The custom duty ...
A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec infusion.
Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for ...
Scholar Rock (SRRK), a late-stage biopharmaceutical company developing innovative treatments for spinal muscular atrophy, ...
Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.
Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...
Filmmaker Reid Davenport weaves a powerful first-person perspective with a journalistic investigation about disability ...
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