“One of [these therapeutics], risdiplam, is a small molecule compound that enhances the splicing and expression of SMN2, a ...

A drug cost analysis expert reveals that the life-saving drug Risdiplam, used to treat the rare genetic disease SMA, could ...

Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for ...

Researchers have uncovered a mechanism that may trigger ALS’s earliest stages, identifying proteins that mislocalize, causing ...

Risdiplam is a potential rival to Biogen’s Spinraza (nusinersen), a spinally injected antisense drug that tells the body to produce the SMN protein that is defective in patients with SMA and has ...

Public health groups are challenging Roche's attempt to block a generic version of its expensive Spinal Muscular Atrophy drug ...

While Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market for spinal muscular atrophy (SMA) treatments, Novartis’ research chief Jay Bradner has a ...

A drug pricing expert reveals the shocking cost disparity of a life-saving spinal muscular atrophy drug in India, priced at ...

Health groups and patients calls for government interference to make rare disease drugs affordable: Our Bureau, New Delhi Tuesday, November 5, 2024, 18:10 Hrs [IST] Health groups ...

Roche, a Swiss multinational healthcare company, is seeking a permanent injunction against Natco Pharma to prevent the introduction of a generic version of Risdiplam, a Spinal Muscular Atrophy (SMA) ...

Patients with rare diseases in India, like Saifullah Khalidi with spinal muscular atrophy (SMA), struggle to afford ...

Roche’s action in Delhi High Court seeking an injunction against Natco Pharma to prevent the introduction of a generic ...