Spinal muscular atrophy (SMA) is a group of genetic disorders that causes a loss of motor nerve cells and muscle atrophy. There are five types of SMA relating to age of onset and symptom severity.

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 ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

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

A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

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

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 ...

The bench said that public good outweighs the company’s profit as the drug is not available at an affordable price in India.

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 ...