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The Neuromuscular Disorders Channel:
Spinal Muscular Atrophy

Welcome to The Spinal Muscular Atrophy Channel

VJNeurology brings you the latest updates in spinal muscular atrophy directly from world-leading experts. The Spinal Muscular Atrophy Channel brings you video interviews from international conferences.

Discover key developments in this space, including updates in disease biology, novel treatment strategies, and ongoing clinical trials.

Risdiplam in infants with genetically diagnosed and presymptomatic SMA: the RAINBOWFISH trial 2:27
Risdiplam in infants with genetically diagnosed and presymptomatic SMA: the RAINBOWFISH trial
Maria Mazurkiewicz-Bełdzińska • 20 Oct 2025
The importance of early intervention in NMDs and challenges that remain 2:05
The importance of early intervention in NMDs and challenges that remain
Massimiliano Filosto • 23 Jun 2025
The shift toward targeted, disease-modifying therapies in neuromuscular disorders 2:36
The shift toward targeted, disease-modifying therapies in neuromuscular disorders
Massimiliano Filosto • 23 Jun 2025
Unmet needs for patients with NMDs and the evolving role of gene therapy 1:57
Unmet needs for patients with NMDs and the evolving role of gene therapy
Michelangelo Mancuso • 22 Jun 2025
Latest regarding SMA biomarkers for disease monitoring and treatment response 0:52
Latest regarding SMA biomarkers for disease monitoring and treatment response
Robert Muni-Lofra • 2 Jul 2023
The changing therapeutic landscape of SMA 1:32
The changing therapeutic landscape of SMA
Robert Muni-Lofra • 2 Jul 2023
The Adult SMA REACH project: a data collection study 1:58
The Adult SMA REACH project: a data collection study
Robert Muni-Lofra • 2 Jul 2023
Using real-world data in SMA management 2:18
Using real-world data in SMA management
Anna Kostera-Pruszczyk • 25 Jun 2022
Treating spinal muscular atrophy in adult patients 3:13
Treating spinal muscular atrophy in adult patients
Anna Kostera-Pruszczyk • 25 Jun 2022
Recommended standards of care for adult patients with spinal muscular atrophy 2:26
Recommended standards of care for adult patients with spinal muscular atrophy
Anna Kostera-Pruszczyk • 25 Jun 2022
Approved gene-based therapies for neuromuscular diseases 3:57
Approved gene-based therapies for neuromuscular diseases
Aida Abu-Baker • 11 Oct 2021
Outcome measures in motor neuron diseases and neuropathies 3:03
Outcome measures in motor neuron diseases and neuropathies
Marianne de Visser • 28 Sep 2021
Novel outcome measures for ALS and SMA 3:26
Novel outcome measures for ALS and SMA
Marianne de Visser • 28 Sep 2021
Exciting developments in the neuromuscular field 1:52
Exciting developments in the neuromuscular field
Gabriele Siciliano • 15 Jun 2021
Apitegromab for the treatment of SMA 2:49
Apitegromab for the treatment of SMA
Gabriele Siciliano • 15 Jun 2021
The progress in gene therapy in neuromuscular diseases since its first clinical use 5:51
The progress in gene therapy in neuromuscular diseases since its first clinical use
Jerry Mendell • 6 Apr 2021
The content of VJNeurology is intended for healthcare professionals