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Spinal muscular atrophy, type II
MONDO:0009673Proximal spinal muscular atrophy type 2 (SMA2) is a chronic infantile form of proximal spinal muscular atrophy characterized by 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: Intermediate spinal muscular atrophy, SMA type 2, SMA type II, SMA-II, SMA2, chronic infantile spinal muscular atrophy, chronic spinal muscular atrophy, muscular atrophy, spinal, infantile chronic form
41 clinical trials for this condition and its sub-types.
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Gene therapy hope for SMA kids: early trial launches
Disease control OngoingThis early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …
Phase: NA • Sponsor: Nikegen Pharmaceutical (Hangzhou) Company Limited • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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SPINRAZA study tracks movement in adults with spinal muscular atrophy
Disease control OngoingThis study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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AI boosts brain scan accuracy in massive new trial
Diagnosis ENROLLING_BY_INVITATIONThis study tests whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…
Sponsor: Yaou Liu • Aim: Diagnosis
Last updated Jun 27, 2026 11:00 UTC
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Could a headset at home boost brain recovery? new study tests tDCS for stroke, tumors, and neurodegeneration
Symptom relief ENROLLING_BY_INVITATIONThis study tests whether a home-based brain stimulation device (tDCS) combined with activity therapy can help improve cognitive and language problems in people with stroke, brain tumors, or neurodegenerative conditions like Parkinson's or Alzheimer's. Fifty-five participants will…
Phase: NA • Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC
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Smart sleeve study aims to improve movement for nerve disease patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study follows 1000 people with upper motor neuron disease who are about to receive the Cionic Neural Sleeve. The sleeve uses electrical stimulation to help muscles contract at the right time during movement. Researchers will track changes in health-related quality of life us…
Phase: NA • Sponsor: Cionic, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:04 UTC