Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

[Gene-based treatment in spinal muscular atrophy].

Der Nervenarzt - 1 Jun 2022

Hagenacker T, Schara-Schmidt U, Kleinschnitz C

Abstract excerpt

BACKGROUND: The 5q-associated spinal muscular atrophy (SMA) affects ~ 80-120 newborns annually. The disease is characterized by progressive paresis involving the bulbar and respiratory musculatures. The phenotypes are very heterogeneous ranging from severe courses with early death in the first years of life to loss of gait in older age. OBJECTIVE: There are now an increasing number of causally targeted therapies...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Gait
Share to Infant, Newborn
Share to Muscular Atrophy, Spinal

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Oligonucleotides

Share to Phenotype

Share to Risk Assessment

Source

Identifiers

PMID: 35522309
DOI: 10.1007/s00115-022-01295-1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

[Gene-based treatment in spinal muscular atrophy].PMID 35522309Read ↗

[SMA: from gene discovery to gene therapy].Semantic proximity Read ↗

Gene therapy in spinal muscular atrophy.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 ↗

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent LandscapeSemantic 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].Semantic proximity Read ↗

New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?Semantic proximity Read ↗

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).Semantic proximity Read ↗

Current and emerging targeted therapies for spinal muscular atrophy.Semantic proximity Read ↗

Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and ChallengesSemantic proximity Read ↗

Spinal muscular atrophy: from gene discovery to clinical trials.Semantic proximity Read ↗

Current advances in drug development in spinal muscular atrophy.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.