In an interview-based study, adults with SMA reported a lack of coordination during their transition from pediatric care once ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

As their 8-month-old daughter prepares for gene therapy treatment, a Coon Rapids family is bracing for three months of ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...

The Rare Disease Therapeutics Market is experiencing an unprecedented wave of innovation, driven by gene therapies, RNA-based ...

Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington's disease, the Swiss drugmaker said on Wednesday, as it plans a clinical ...

The Muscular Dystrophy Association (MDA) today kicked off its largest annual MDA Holiday Retail Campaign, spanning thousands of retail ...

The Delhi High Court has dismissed Roche's appeal, allowing Natco to launch a generic version of Risdiplam for spinal ...

Scholar Rock (SRRK) stock gains as Bank of America launches its coverage with a Buy recommendation, citing a blockbuster potential for its lead drug. Read more here.