Nov 8, 2024 · Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according to a study.

Oct 30, 2024 · Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with SMA, a study found.

Sep 13, 2024 · Real-world treatment with Zolgensma improves motor function in SMA children who carry at least four copies of the so-called backup SMN2 gene.

Oct 21, 2024 · In the absence of treatment, the long-term progression of spinal muscular atrophy (SMA) types 2 and 3 is highly variable and seems to be influenced by factors including a …

Oct 11, 2024 · A high-dose regimen of SMA treatment Spinraza benefits for previously treated and untreated SMA patients of all ages, per trial data.

Dec 18, 2024 · SMA type 4 is the mildest and one of the rarest forms of the disease. Learn more about symptoms, diagnosis, and treatment options.

Dec 4, 2024 · Zolgensma was found in a real-world study to ease breathing difficulties in children with SMA and reduce respiratory-related hospitalizations.

Jan 8, 2025 · The addition of the protein, raising SMN protein levels (expression), in combination with an SMN2 -targeting therapy was more effective than either of those approaches alone, …

Oct 18, 2024 · Adults with spinal muscular atrophy (SMA) showed alterations in body composition and metabolic disturbances, including changes in blood sugar and fats, that could leave them …

6 days ago · Type 2 is an intermediate form of spinal muscular atrophy (SMA), a rare genetic disease characterized by the progressive loss of motor neurons — the nerve cells controlling …