Apitegromab hits goal of boosting motor function in Phase 3 trial
Oct 9, 2024 · The SAPPHIRE study enrolled 188 young adults and children with SMA type 2 or 3, including 156 patients, ages 2-12, in the main group, and 32 patients, ages 13-21, in an …
Apitegromab (SRK-015) for spinal muscular atrophy - SMA News …
Jul 18, 2024 · Apitegromab in clinical trials. Apitegromab has been tested in a Phase 1 study in healthy volunteers which showed positive safety results and indicated the therapy was …
Zolgensma most successful in presymptomatic SMA infants: Study
Nov 8, 2024 · Zolgensma delivers working copies of SMN1 to body’s cells. Zolgensma (onasemnogene abeparvovec-xioi) is a one-time gene therapy that delivers working copies of …
Top 10 SMA news stories of 2024
Jan 3, 2025 · Read this story here. No. 5 — Combination therapy found to synergistically boost SMN production. When combined with a therapy like Spinraza, low-dose risdiplam — the …
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
Feb 2, 2023 · 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 …
SMA treatment benefits increase after Spinraza-Zolgensma switch
Nov 18, 2024 · Some 70% of children with spinal muscle atrophy (SMA) type 1 who underwent treatment with Spinraza alone or with Spinraza and then Zolgensma reached some motor …
High dose of SMA treatment Spinraza shows benefits: Trial data
Oct 11, 2024 · Data expected to support regulatory applications come from a pivotal group of patients enrolled in the study’s Part B. This involved 75 symptomatic infants with SMA, ages …
Unique BICD2 mutation causes severe SMA-LED: Case study
Dec 4, 2024 · The case study, “ A Novel De Novo Splice Acceptor Variant in BICD2 Is Associated With Spinal Muscular Atrophy,” was published in the American Journal of Medical Genetics. …
Upcoming Events – SMA News Today
This Engage Symposium will provide updates on all neuromuscular diseases including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), amyotrophic lateral sclerosis …
SMA treatment Spinraza eases fatigability in patients who can walk
Oct 30, 2024 · The study, “ Performance fatigability in adults with spinal muscular atrophy treated long-term with nusinersen,” was published in Muscle and Nerve. SMA is marked by symptoms …