in patients aged 2 to less than 18 years with spinal muscular atrophy (SMA) type 2. The randomized, double-blind, sham-controlled phase 3 STEER study (ClinicalTrials.gov Identifier: NCT05089656 ...

Back to Healio Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease ...

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a ...

in treating individuals with spinal muscular atrophy (SMA) Type 2. The randomised, sham-controlled study assessed the efficacy and safety of the gene therapy in treatment-naïve individuals aged ...

More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...

The discussions mainly focused on apitegromab for spinal muscular atrophy (SMA), including ... which involves patients with non-ambulatory type 2/3 SMA treated with Spinraza (nusinersen) or ...

One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder affecting ... Symptoms of SMA can vary widely depending on the specific type and the age of onset. The most prevalent ...

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...