Spinal muscular atrophy, type 1
MONDO:0009669A severe infantile form of proximal spinal muscular atrophy characterized by severe and progressive muscle weakness and hypotonia resulting from the degeneration and loss of the lower motor neurons in the spinal cord and the brain stem nuclei.
Also known as: infantile muscular atrophy, infantile spinal muscular atrophy, spinal muscular atrophies of childhood, SMA type 1, SMA type I, SMA-I, SMA1, SMNI
32 clinical trials for this condition and its sub-types, 17 tagged with Spinal muscular atrophy, type 1 itself.
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Gene therapy hope for SMA kids: early trial launches
Disease control OngoingThis early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …
Sponsor: Nikegen Pharmaceutical (Hangzhou) Company Limited • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Gene therapy hope for babies with fatal muscle disease
Disease control OngoingThis study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy …
Sponsor: Kun Sun • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC