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 …
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. …
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 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 …
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 …
Raising SMN protein levels may boost SMA treatment effectiveness
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, the …
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, …
MDA 2024: Zolgensma gene therapy safe in children up to 21 kg
Mar 6, 2024 · That’s according to the poster, “ Safety and Efficacy of Intravenous Onasemnogene Abeparvovec in Pediatric Patients with Spinal Muscular Atrophy: Findings from the Phase 3b …
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 …
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 …