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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One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own
Disease control CompletedThis phase 3 trial tested a one-time gene therapy called Zolgensma in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN gene via an IV infusion. The main goals were …
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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One-Time gene therapy helps babies with rare muscle disease sit on their own
Disease control CompletedThis study tested a one-time gene replacement therapy in infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The main goal was to see if treated babies could sit without support for at least 10 seconds by 18 months of age. The …
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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One-Time gene therapy helps babies with fatal muscle disease sit independently
Disease control CompletedThis study tested a one-time gene replacement therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment aims to replace the missing SMN1 gene to improve muscle function. Key results showed that many babies…
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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Gene therapy breakthrough: one dose may help babies with rare muscle disease
Disease control CompletedThis study tested a single dose of Zolgensma gene therapy in 30 infants diagnosed with spinal muscular atrophy (SMA) before symptoms appeared. The goal was to see if the treatment helps them reach motor milestones like sitting or standing alone. The therapy delivers a working cop…
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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Laser test could objectively measure nerve pain for first time
Diagnosis CompletedThis study tested whether a diode laser can act as a biomarker to measure neuropathic pain in people with peripheral neuropathy. Researchers compared pain responses to a lidocaine patch versus a placebo patch in 75 participants. The goal was to see if the laser test could disting…
Sponsor: University of Utah • Aim: Diagnosis
Last updated Jun 27, 2026 12:09 UTC
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Can a 12-Week online course help babies with SMA develop better?
Symptom relief CompletedThis study tests a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…
Sponsor: Medipol University • Aim: Symptom relief
Last updated Jul 11, 2026 00:00 UTC
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Study examines how to better support families of infants with fatal muscle disease
Knowledge-focused CompletedThis study looked at the quality of supportive and palliative care for infants under one year old with spinal muscular atrophy (SMA) type 1, a severe genetic muscle disease. Researchers followed 39 infants and asked families to keep a diary about care. One year after the child's …
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:33 UTC