SPINAL MUSCULAR ATROPHY 1
Clinical trials for SPINAL MUSCULAR ATROPHY 1 explained in plain language.
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New gene therapy aims to help babies with deadly muscle disease
Disease control Not yet recruitingThis early-stage study tests a new medicine called SKG0201 in 11 babies with spinal muscular atrophy type I, a severe genetic condition that causes muscle weakness. The main goals are to check if the treatment is safe and to see if it can help babies survive and develop motor ski…
Matched conditions: SPINAL MUSCULAR ATROPHY 1
Phase: PHASE1, PHASE2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated Jun 16, 2026 12:52 UTC
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What do families of kids with SMA type 1 go through? new study asks parents directly.
Knowledge-focused Not yet recruitingThis study will interview 15 parents or guardians of children with spinal muscular atrophy type 1 (SMA1) to learn about their daily experiences with feeding and communication. Even though newer medicines have improved survival, how they affect swallowing and speech is still uncle…
Matched conditions: SPINAL MUSCULAR ATROPHY 1
Sponsor: Guy's and St Thomas' NHS Foundation Trust • Aim: Knowledge-focused
Last updated Jun 15, 2026 18:54 UTC