In a case that was billed as profits versus patients, India’s Supreme Court dismissed a petition in which Roche sought to ...

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

Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...

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 ...

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

The Neuromuscular Disease Therapeutics Market is entering a period of accelerated growth, supported by breakthroughs in gene ...

The Supreme Court upheld Delhi HC's interim order allowing Natco Pharma to manufacture and sell a generic version of Roche's ...

For people with spinal muscular atrophy, physical therapy can help support mobility, strength, and quality of life, regardless of SMA type.

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

In a huge setback for Swiss pharma major Roche, the Supreme Court on Friday dismissed its petition challenging a Delhi High ...

Roche has moved the Supreme Court challenging a Delhi HC order that allowed Natco Pharma to manufacture and sell a generic ...

The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...