A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...

The Muscular Dystrophy Association celebrates this new more accessible treatment option for people living with SMA. MDA - New ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stock has reached a new 52-week low, touching down at $30.86, with InvestingPro data showing the stock trading at $30.89, significantly below its 52-week high ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...

Biotech giant Biogen Inc (NASDAQ:BIIB) is scheduled to report fourth-quarter earnings on 12 February. The Wall Street ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stock has touched a 52-week low, dipping to $31.24, as investors respond to a challenging year for the biotechnology firm. The company, known for its ...

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 ...

The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months. The FDA has accepted a supplemental ...

The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved ...

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are now reviewing the supplemental New Drug Application (sNDA) for nusinersen, which includes a more rapid ...

Biogen Inc. announced that the FDA has accepted and the EMA has validated its supplemental New Drug Application for a higher dose regimen of nusinersen, intended for treating spinal muscular ...