Spinal Muscular Atrophy Type 2 Latest Advances

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

Journal: JAMA network open

Published: October 08, 2025

Reviewer Comment on Gauvreau et al. "Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen".

Journal: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques

Published: October 03, 2025

Results in our symptomatic and presymptomatic SMA patients treated with disease-modifying therapy

Journal: Ideggyogyaszati szemle

Published: October 01, 2025

A sensory and motor neuropathy caused by a genetic variant of NAMPT.

Journal: Science advances

Published: September 26, 2025

Isolated peripheral neuropathy in a young adult: the role of comprehensive genetic testing.

Journal: BMJ case reports

Published: September 19, 2025

Co-occurrence of ipsilateral partial Horner's syndrome in a patient with monomelic amyotrophy.

Journal: BMJ case reports

Published: September 05, 2025

Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.

Journal: Journal of neuromuscular diseases

Published: September 03, 2025

The impact of early powered mobility training in families and children with Spinal Muscular Atrophy Type I.

Journal: Assistive technology : the official journal of RESNA

Published: August 22, 2025

Hirayama Disease in a Young Male: A Case Report.

Journal: JNMA; journal of the Nepal Medical Association

Published: July 14, 2025

Hereditary neuropathy with liability to pressure palsies with recurrent facial paralysis as main clinical manifestation in a Chinese patient: A case report.

Journal: Medicine

Published: July 09, 2025

Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3.

Journal: European journal of pediatrics

Published: July 02, 2025

Neuromuscular pathology and mitochondrial dysfunction in sorbitol dehydrogenase gene-related distal hereditary motor neuropathies.

Journal: Journal of neuropathology and experimental neurology

Published: June 28, 2025

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Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

Reviewer Comment on Gauvreau et al. "Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen".

Reviewer Comment on Gauvreau et al. "Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen".

Results in our symptomatic and presymptomatic SMA patients treated with disease-modifying therapy

Results in our symptomatic and presymptomatic SMA patients treated with disease-modifying therapy

A sensory and motor neuropathy caused by a genetic variant of NAMPT.

A sensory and motor neuropathy caused by a genetic variant of NAMPT.

Isolated peripheral neuropathy in a young adult: the role of comprehensive genetic testing.

Isolated peripheral neuropathy in a young adult: the role of comprehensive genetic testing.

Co-occurrence of ipsilateral partial Horner's syndrome in a patient with monomelic amyotrophy.

Co-occurrence of ipsilateral partial Horner's syndrome in a patient with monomelic amyotrophy.

Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.

Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.

The impact of early powered mobility training in families and children with Spinal Muscular Atrophy Type I.

The impact of early powered mobility training in families and children with Spinal Muscular Atrophy Type I.

Hirayama Disease in a Young Male: A Case Report.

Hirayama Disease in a Young Male: A Case Report.

Hereditary neuropathy with liability to pressure palsies with recurrent facial paralysis as main clinical manifestation in a Chinese patient: A case report.

Hereditary neuropathy with liability to pressure palsies with recurrent facial paralysis as main clinical manifestation in a Chinese patient: A case report.

Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3.

Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3.

Neuromuscular pathology and mitochondrial dysfunction in sorbitol dehydrogenase gene-related distal hereditary motor neuropathies.

Neuromuscular pathology and mitochondrial dysfunction in sorbitol dehydrogenase gene-related distal hereditary motor neuropathies.