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

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

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 Delhi High Court on Monday dismissed an interim injunction application filed by Swiss pharmaceutical giant F Hoffmann-La ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Video: Spinal Muscular Atrophy: When is Surgical Intervention Appropriate? —How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit ...

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

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

The high court noted that SMA is a debilitating disease which has no cure, and the only approved drug, Risdiplam, marketed ...

—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 ...

Innovator drug price is Rs 22-72 lakh per year; Analysts forecast Natco to price generic drug at Rs 3,000 per year ...

In a major relief to Indian drugmaker Natco Pharma, the Delhi High Court has rejected an interim injunction plea filed by ...