autosomal recessive distal spinal muscular atrophy 1

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
UK SMA Patient Registry	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Spinal Muscular Atrophies of Childhood, X-linked disease, autosomal recessive distal spinal muscular atrophy 1, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy	Recruiting	Not stated	2026-08-20		NCT04292574
Gene Therapy for IGHMBP2-Related Diseases	Charcot-Marie-Tooth disease axonal type 2S, autosomal recessive distal spinal muscular atrophy 1	Enrolling by invitation	Phase 1 / Phase 2	2025-09-30		NCT05152823

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