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 ...

Evrysdi is the third treatment approved for SMA, an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma ...

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

and Tysabri (natalizumab), as well as blockbuster spinal muscular atrophy therapy Spinraza (nusinersen). Biogen will retain its commercial rights to the biosimilars already brought to market ...

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 ...

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

Introduction: There is continued unmet medical need for new medicines across countries especially for cancer, immunological diseases, and orphan diseases. However, there are growing challenges with ...

Cotización tras cierre: January 31 at 6:56:40 PM EST Loading Chart for IONS ...