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

Modern approaches in gene therapy of motor neuron diseases

8 Jul 2020

Abstract excerpt

Motor neuron disorders are a group of neurodegenerative diseases characterized by muscle weakness, loss of ambulation, respiratory insufficiency, leading to an early death. Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis are the most common and fatal motor neuron diseases. The last 3 years became very successful for novel gene therapy approaches in SMA in infants. Two innovative drugs-nusinersen...

Read the complete abstract on PubMed

Topics

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

Share to Muscle Physiology and Disorders
Share to Genetics and Neurodevelopmental Disorders
Share to Neurogenetic and Muscular Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 32638429
DOI: 10.1002/med.21705

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Modern approaches in gene therapy of motor neuron diseasesPMID 32638429Read ↗

Gene Therapy in ALS and SMA: Advances, Challenges and PerspectivesSemantic proximity Read ↗

Bridging the Gap: Gene Therapy in a Patient With Spinal Muscular Atrophy Type 1Semantic proximity Read ↗

Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disordersSemantic proximity Read ↗

Gene‐based treatment of motor neuron diseasesSemantic proximity Read ↗

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

Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophySemantic proximity Read ↗

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

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

Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral SclerosisSemantic proximity Read ↗

Significance of gene therapy in neurodegenerative diseasesSemantic proximity Read ↗

Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMASemantic proximity Read ↗

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

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