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 ...

Patrick R. O'Neil, Executive Vice President, Chief Legal Officer & General Counsel of Ionis Pharmaceuticals Inc. (NASDAQ:IONS), recently sold 6,165 shares of the company's common stock. The shares ...

Brett P. Monia, the Chief Executive Officer of Ionis Pharmaceuticals Inc. (NASDAQ:IONS), sold 38,843 shares of the company's common stock on February 4, according to a recent SEC filing. The sale ...

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 ...

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 ...

The FDA recently approved Vertex Pharmaceuticals Incorporated’s Journavx (suzetrigine), a first-in-class non-opioid ...

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Pre-Market: 4:19:35 am GMT-5 ...

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

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 ...

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