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 ...

Biogen (NASDAQ:BIIB) shares experienced a change in market expectations as Cantor Fitzgerald revised the company's price target. The new target has been set at $206.00, a decrease from the previous ...

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

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 ...

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.

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 ...

The use of long-term ventilation (LTV) in children is growing in the UK and worldwide. This reflects the improvement in technology to provide LTV, the growing number of indications in which it can be ...