A mass recruitment has been underway for more desperately-needed nurses for the NHS following the death in hospital of a woman with ...

The symptoms of myotonic dystrophy may be obvious from birth or they can develop later -- during the teenage or adult years. Like other forms of muscular dystrophy, myotonic dystrophy leads to ...

Q1 2025 Earnings Call Transcript February 10, 2025 Arrowhead Pharmaceuticals, Inc. misses on earnings expectations. Reported EPS is $-1.39 EPS, expectations were $-0.42. Chris Anzalone: Thanks, Vince.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has ...

The U.S. Food and Drug Administration approved twice-daily JOURNAVX for the treatment of adults with moderate-to-severe acute ... type 1 diabetes and myotonic dystrophy type 1. This press release ...

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic dystrophy type ... a month among older adults, with longer wait ...

Methods: 598 patients with a neuromuscular disease were studied (139 with facioscapulohumeral dystrophy, 322 with adult onset myotonic dystrophy, and 137 with hereditary motor and sensory neuropathy ...

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® ...

The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1 ... vein dosing regimens of DYNE-101 against a placebo in ...