SPINAL MUSCULAR ATROPHY 1
Clinical trials for SPINAL MUSCULAR ATROPHY 1 explained in plain language.
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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…
Matched conditions: SPINAL MUSCULAR ATROPHY 1
Phase: PHASE1, PHASE2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:05 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…
Matched conditions: SPINAL MUSCULAR ATROPHY 1
Sponsor: Guy's and St Thomas' NHS Foundation Trust • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:00 UTC