Article

SPINAL MUSCULAR ATROPHY: CLINICAL CHARACTERIZATION AND EVOLUTION AFTER TREATMENT WITH NEW MODIFYING THERAPIES

2025-02-13

Abstract excerpt

<h4>Objectives: </h4> Conducting a retrospective analysis of the clinical characteristics and evolution of patients with spinal muscular atrophy (SMA) type 1 and 2 which were treated with the new modifying therapies. <h4>Methods:</h4> A descriptive, cross-sectional and retrospective study was conducted with a target population of patients with a genetic diagnosis of SMA treated with nusinersen or onasemnogene abep...

Topics

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

Identifiers and source

Literature Corpus work: b8b92307-863f-5172-9080-36d2898c70ad
DOI: 10.22541/au.173941035.57379117/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

SPINAL MUSCULAR ATROPHY: CLINICAL CHARACTERIZATION AND EVOLUTION AFTER TREATMENT WITH NEW MODIFYING THERAPIESDOI 10.22541/au.173941035.57379117/v1Read

The respiratory impact of novel therapies for spinal muscular atrophy.Semantic proximity Read

The Effects of Nusinersen Treatment on Respiratuar Status of Children with Spinal Muscular AtrophySemantic 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

Spinal muscular atrophy: A changing phenotype beyond the clinical trials.Semantic proximity Read

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

The efficacy of gene therapy with onasemnogene abeparvovec in spinal muscular atrophy in young patientsSemantic proximity Read

Real world safety and exploratory efficacy of gene therapy for patients with 5q-Spinal Muscular Atrophy in a Brazilian cohortSemantic proximity Read

Effect of nusinersen after three years of treatment in 61 young children with SMA type 1 or 2: a French real-life observational studySemantic proximity Read

Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective.Semantic proximity Read

Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophySemantic proximity Read

A self-reported Brazilian registry of spinal muscular atrophy: data from natural history, genetic characteristics, and multidisciplinary careSemantic proximity Read

Current understanding of and emerging treatment options for spinal muscular atrophy with respiratory distress type 1 (SMARD1)Semantic proximity Read