Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

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 ...

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

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

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

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 ...

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 ...

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

Karnataka launched free treatment for Spinal Muscular Atrophy (SMA) at the Indira Gandhi Institute of Child Health in ...

Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...