Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP. The positive opinion ...

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 euros, and ...

Session ID: 2025-07-03:66e5703a8f0ad03a5f75bd6c Player Element ID: vjs_video_3 ...

Spinal Muscular Atrophy treatment is evolving fast, with gene therapy, oral drugs, and next-gen muscle enhancers offering new hope and long-term outcomes. With each breakthrough in SMA treatment ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that its pivotal dose improves functional outcomes in older boys with the disease.

Leaders from the world of cell and gene therapy, including molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates, shared their personal stories and policy ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy.

Rs 16 crore gene therapy hope for 16-month-old: Parents raise Rs 9 crore for rare disease The baby from Ranaghat in Nadia is suffering from spinal muscular atrophy (SMA) Type I, a condition that ...

Researchers at the University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease that could help protect the brain from damage and preserve cognitive ...

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease that could help protect the brain from damage and preserve cognitive ...

FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy At roundtable, Kennedy, Prasad, and others express openness to quick approvals Adobe By Jason Mast June 6, 2025 ...

Scientists from the University of California, San Diego (UC San Diego), have published data from a study that tested the effects of a gene therapy for Alzheimer’s disease (AD) dubbed AAV9 ...