Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

OAV101 IT is an investigational, one-time gene therapy for patients with SMA. In the registrational phase III STEER study, OAV101 IT treatment resulted in a statistically significant 2.39-point ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of genetic ...

FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

SMA patients who can sit see improvements in motor function over 12 months of treatment with DMTs, particularly in fine motor skills.

The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and ...

Affordable generic version of SMA drug Risdiplam soon available in India, offering hope to patients and activists.

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 ...

Key to that mission is the “STEER” study, a late-stage trial of children with moderate SMA who are older than ... infection for the sham group. Many gene therapies use adeno-associated viruses ...

Back in 2022, Novartis recorded two deaths from acute liver failure after patients were treated with its spinal muscular atrophy gene therapy Zolgensma. For Elevidys, the deceased patient also had ...