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 ...

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

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

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 ...

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

Dennis P. Scanlon, PhD; Emma Ciafaloni, MD; Maria Lopes, MD, MS; Kevin U. Stephens, Sr, JD, MD; and Mary Schroth, MD, FAAP, FCCP provide an overview of the disease burden in spinal muscular atrophy ...

Biogen's Spinraza is not the first drug to face a CMC-related rejection recently, as SMA competitor drug, apitegromab, also got the FDA no-go ...

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 ...