FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

Learn more about whether Ionis Pharmaceuticals, Inc. or Moderna, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Hirayama disease is a rare neurological disorder primarily affecting young males. This condition poses diagnostic challenges ...

Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

On Wednesday, 919 stocks advanced, 3115 declined and 109 remained unchanged, with an advance decline ratio of 0.29 on the Bombay Stock Exchange (BSE), indicating negative closing of stocks in broader ...

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

Results from the STEER and STRENGTH studies showed that OAV101 IT led to a 2.39-point improvement in motor function in patients with spinal muscular atrophy. Results from the Phase III STEER and Phase ...

Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad group of patients aged 2 to ...

—Sleep-disordered breathing occurs frequently in patients with spinal muscular atrophy and can be obstructive or non-obstructive, with those diagnosed with non-obstructive SDB having worse ...

Novartis has announced positive results from a late-stage programme of its experimental gene replacement therapy in patients with the rare neuromuscular disease spinal muscular atrophy (SMA). The ...