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 ...

Biogen has announced that the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have started ...

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

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 ...

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA ... after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stock has touched a 52-week low, dipping to $31.24, as investors respond to a ...

Novartis' programme providing free access to its spinal muscular atrophy (SMA ... to market its antisense-based therapy Spinraza (nusinersen) since 2016, while Roche got a green light for its ...

In surveys, parents of children with SMA and healthcare professionals in Sweden note the many ways the pandemic affected ...

So the outcome and the long-term outlook for our patients with SMA, particularly in the adult populations are that they are now stronger and living longer. And so as a result of that, they're able ...

A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec infusion.

Scholar Rock is asking the U.S. Food and Drug Administration to approve apitegromab for people with spinal muscular atrophy.