Please sign in to follow a disease.
Spinal muscular atrophy, type 1
MONDO:0009669A severe infantile form of proximal spinal muscular atrophy characterized by severe and progressive 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: infantile muscular atrophy, infantile spinal muscular atrophy, spinal muscular atrophies of childhood, SMA type 1, SMA type I, SMA-I, SMA1, SMNI
45 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsBroader categories
-
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
-
Gene therapy hope for babies with fatal muscle disease
Disease control OngoingThis study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy …
Phase: NA • Sponsor: Kun Sun • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
-
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
-
Can a 12-Week online course help babies with SMA develop better?
Symptom relief OngoingThis study tests a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…
Phase: NA • Sponsor: Medipol University • Aim: Symptom relief
Last updated Jun 27, 2026 12:23 UTC
-
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
-
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