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

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 ...

A 16-month-old boy from a low-income family in Kolkata receives a rare gene therapy for spinal muscular atrophy free of ...

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

SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do no ...

The study analyzed outcomes against the number of copies of the SMN2 gene each child had ... including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder ...

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 ...

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 ...

The five-year award will be used to consider the genetic and cellular determinants that may be limiting the success of new gene therapies ... treatment of spinal muscular atrophy, inherited ...

Treatment options for Spinal Muscular Atrophy (SMA), a rare genetic disease, are virtually nonexistent in India, primarily ...

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 ...