for apitegromab for the improvement of motor function in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 weeks compared with sham in patients aged 2 to 18 years with spinal muscular ...

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 ...

Purpose: X-linked infantile spinal-muscular atrophy (XL-SMA) is a rare disorder, which presents with the clinical characteristics of hypotonia, areflexia, and multiple congenital contractures ...

Evidence suggests inbreeding heightens the risk of birth defects and developmental delays, such as learning and speech ...

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for apitegromab for the improvement of motor function in patients with spinal muscular ...

Spinal muscular atrophy (SMA) is a disorder affecting the motor neurons—nerve cells that control voluntary muscle movement. These cells are located in the spinal cord. Because the muscles cannot ...

22, 2025. Scholar Rock announced today that the FDA granted priority review for a biologics license application (BLA) for apitegromab, a treatment for spinal muscular atrophy (SMA), according to a ...

an oral medicine for Spinal Muscular Atrophy (SMA). Loading content, please wait ...

A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and often leads to infant mortality In a breakthrough for Spinal Muscular ...