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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Can gene therapy help babies with SMA reach milestones? a Real-World review
Disease control Not yet recruitingThis study looks back at medical records of children with spinal muscular atrophy (SMA) type 1 who received a one-time gene therapy called onasemnogene abeparvovec (Zolgensma). Researchers want to see if treated infants can sit independently for 30 seconds or more within a year. …
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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Horse power: new therapy hopes to improve movement in SMA children
Symptom relief Not yet recruitingThis study tests whether a special type of horseback physiotherapy can improve movement, posture, breathing, and quality of life in children with spinal muscular atrophy (SMA). Twenty children aged 2 to 9 years will receive both the horse therapy and standard physiotherapy in ran…
Sponsor: Charles University, Czech Republic • Aim: Symptom relief
Last updated Jun 27, 2026 08:10 UTC
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New study explores how families cope with feeding and talking challenges in SMA type 1
Knowledge-focused Not yet recruitingThis study will interview 15 parents or guardians of children with spinal muscular atrophy type 1 to learn about their daily challenges with feeding and communication. Even though newer treatments have improved survival, their impact on swallowing and speech is not well understoo…
Sponsor: Guy's and St Thomas' NHS Foundation Trust • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:00 UTC
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New study probes emotional toll of SMA screening on new parents
Knowledge-focused Not yet recruitingThis study looks at how parents feel after their newborn is screened for spinal muscular atrophy (SMA). Researchers will interview 36 parents in two French regions to understand their anxiety, stress, and support needs. The goal is to improve how screening results are shared and …
Sponsor: University Hospital, Strasbourg, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:54 UTC