Article

The efficacy of gene therapy with onasemnogene abeparvovec in spinal muscular atrophy in young patients

2025-04-30

Abstract excerpt

Introduction. The advent of pathogenetic therapy has significantly changed the prognosis for spinal muscular atrophy (SMA). However, our knowledge of the pathogenetic methods of treating SMA is mostly based on the results of clinical trials, which are largely limited due to the narrow criteria for selecting patients and the short duration of the following-up. Objective. To evaluate the efficacy of onasemnogene abe...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 6733f880-be0c-5ee3-a9d6-5f1bd26c05ce
DOI: 10.46563/2686-8997-2025-6-1-13-25

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

The efficacy of gene therapy with onasemnogene abeparvovec in spinal muscular atrophy in young patientsDOI 10.46563/2686-8997-2025-6-1-13-25Read

Onasemnogene abeparvovec-xioi: a gene replacement strategy for the treatment of infants diagnosed with spinal muscular atrophySemantic proximity Read

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.Semantic proximity Read

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)Semantic proximity Read

New Treatments in Spinal Muscular Atrophy: Positive Results and New ChallengesSemantic proximity Read

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1Semantic proximity Read

Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy.Semantic proximity Read

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the three first years of treatment.Semantic proximity Read

Gene-Based Therapy for the Treatment of Spinal Muscular Atrophy Types 1 and 2 : A Systematic Review and Meta-AnalysisSemantic proximity Read

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence.Semantic proximity Read

Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular AtrophySemantic proximity Read

Therapeutic Efficacy and Safety of onasemnogene abeparvovec Gene Therapy for Spinal Muscular Atrophy Type 1: Updated Systematic Review with Meta-AnalysisSemantic proximity Read

Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular AtrophySemantic proximity Read