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 …
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 …
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 Zolgensma seen to benefit patients in real world
Sep 13, 2024 · The team analyzed data from 19 children — more than half of them boys — meeting these criteria, who were participating in RESTORE (NCT04174157), a real-world, …
SMA breathing difficulties eased with Zolgensma in real-world study
Dec 4, 2024 · Daily ventilation time for Zolgensma-treated children dropped by 3 hours. SMA is caused primarily by mutations in the SMN1 gene, which contains the genetic code for the …
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 …
SMA progression in types 2 and 3 varies without treatment: Study
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 …
SMA Type 4: Symptoms, Treatment, and More - SMA News Today
Dec 18, 2024 · One of the rarest forms of this rare disease, SMA type 4 accounts for fewer than 5% of all cases. It is also called adult-onset or late-onset SMA, because symptoms appear at a …
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 …
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 …