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

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

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

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

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

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.

Former England rugby captain Lewis Moody has been diagnosed with motor neurone disease (MND), a progressive condition ...

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