Gene therapy for Spinal Muscular Atrophy (SMA) is prohibitively expensive in India, with the life-saving drug Zolgensma ...

Kolkata: A 16-month-old child with spinal muscular atrophy (SMA) was chosen for gene therapy and was administered the drug at a private hospital off EM Bypass on Monday. The drug used for the ...

Roche’s Evrysdi (risdiplam) is the market leader among the drugs for spinal muscular atrophy (SMA). Biogen’s Spinraza ...

We are working to raise funds for them through crowdfunding.” Zolgensma (onasemnogene abeparvovec-xioi) is a groundbreaking ...

ACTX-401: Alcyone Therapeutics ACTX-401 is an AAV9-delivered gene replacement therapy for the treatment of a rare form of Spinal Muscular Atrophy (SMA) called SMA with Respiratory Distress type 1 ...

The extension of the TOPAZ study provided support for the long-term benefits and safety of apitegromab therapy in patients with spinal muscular atrophy (SMA) at 36 months. Results from a single ...

Despite diagnostic and therapeutic advances, mortality and the multi-systemic health impact of SMA continue to be experienced ...

After forming a gene therapy partnership with Dyno Therapeutics ... for a rare eye disease and Novartis’ Zolgensma for spinal muscular atrophy, a neurological disorder. Existing AAV vectors ...

MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ: MNOV) and the Standard Market of ...

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 eu ...

These updates will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy ... including Novartis' Zolgensma ® for children with spinal muscular atrophy. Designed to ...