Autosomal dominant childhood-onset proximal spinal muscular atrophy without contractures
MONDO:0008026Also known as: Lower extremity-predominant autosomal dominant proximal spinal muscular atrophy without contractures, SMALED1, spinal muscular atrophy, lower extremity-predominant 1, AD, Kugelberg-Welander syndrome, autosomal dominant, Sma-led, spinal muscular atrophy, LOWER extremity-predominant, 1, autosomal dominant, spinal muscular atrophy, childhood, proximal, autosomal dominant, spinal muscular atrophy, juvenile, proximal, autosomal dominant
29 clinical trials for this condition and its sub-types.
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Could a zapping cap boost memory? small study tests brain stimulation for cognitive decline
Symptom relief Not yet recruitingThis pilot study will test whether a non-invasive brain stimulation technique called transcranial alternating current stimulation (tACS) can improve working memory in 30 adults with mild cognitive impairment or traumatic brain injury. Participants will receive low-level electrica…
Phase: NA • Sponsor: Sunnybrook Health Sciences Centre • Aim: Symptom relief
Last updated Jun 27, 2026 12:28 UTC
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Gene therapy readiness check: new study maps virus immunity in muscle disease patients
Knowledge-focused Not yet recruitingThis study will test blood samples from 450 people aged 6 to 60 with inherited neuromuscular diseases to see if they have antibodies that could block gene therapy viruses (AAVs). The goal is to understand how common these antibodies are and which patients might be good candidates…
Phase: NA • Sponsor: Genethon • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:04 UTC