Progressive muscular atrophy (PMA) is clinically characterized ... in ALS included scores on the revised ALS Functional Rating Scale (ALSFRS-R), and pulmonary forced vital capacity.

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

Spinal muscular atrophy (SMA) is a rare genetic disorder in which the onset of progressive neurodegeneration begins before birth, owing to loss of the protein SMN. Drugs such as risdiplam ...

Age-related muscular atrophy in skeletal muscle can have a devastating ... perspective but are hardly a complete solution given the scale and immediacy of this societal issue.

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

The Phase 3 trial of apitegromab showed statistically significant improvement in motor function as measured by the Hammersmith Functional Motor Scale ... Spinal muscular atrophy is a rare genetic ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

Novartis has announced positive results from a late-stage programme of its experimental gene replacement therapy in patients with the rare neuromuscular disease spinal muscular atrophy (SMA). The ...

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 ...

Try it free for 7 days In the Phase 3 STEER study, treatment with OAV101 IT led to a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), an ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA).

for apitegromab for the improvement of motor functions in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...