Proximal spinal muscular atrophy
MONDO:0019079Proximal spinal muscular atrophies are a group of neuromuscular disorders characterized by progressive muscle weakness resulting from the degeneration and loss of the lower motor neurons in the spinal cord and the brain stem nuclei.
Also known as: SMA, Spinal Muscular Atrophy
54 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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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 Aug 01, 2026 00:00 UTC
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Gene therapy hope for babies with fatal muscle disease
Disease control Not yet recruitingThis early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can…
Phase: PHASE1, PHASE2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:05 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…
Phase: NA • 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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Hidden fracture risk in kids with SMA under spotlight
Knowledge-focused Not yet recruitingThis study looks at bone health in children with spinal muscular atrophy (SMA), a condition that causes muscle weakness. Researchers want to find out how common fractures are and whether newer SMA treatments affect bone strength. They will review medical records from about 550 ch…
Sponsor: Sheffield Children's NHS Foundation Trust • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 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