If not treated, SMA type 1 (SMA-1), the most common and severe form, results in progressive muscle weakness that leads to death. Currently, treatments for SMA-1 have demonstrated improved survival ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...

It occurs due to genetic changes and results in degeneration of the lower motor neurons, causing muscle weakness, muscle atrophy ... for people to receive prompt treatment to manage their symptoms.

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

Treatment with risdiplam is usually ... and even after 30 months had normal muscle development with no sign of atrophy. “During the course of the assessment, we really have seen no indication ...

A new study details this breakthrough case of prenatal therapy that holds promise for future treatment of the neurodegenerative disorder, which leads to the wasting of muscles over time and is usually ...

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve their quality of life.

Following the first-ever treatment for spinal muscular atrophy in the womb ... set in motion before birth that causes worsening muscle weakness. There are four types of the disorder, each with ...