NCT06027385 Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy Status COMPLETED Phase NA
NCT05994950 A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022 Status RECRUITING Phase N/A
NCT05931211 Motor Function and Activity of Daily Life in Spinal Muscular Atrophy Status UNKNOWN Phase N/A
NCT05866939 Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA Status UNKNOWN Phase N/A
NCT05866419 Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy Status RECRUITING Phase NA
NCT05824169 Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients Status RECRUITING Phase PHASE1/PHASE2
NCT05794139 Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy Status ACTIVE_NOT_RECRUITING Phase PHASE2
NCT05769465 MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy Status RECRUITING Phase N/A
NCT05768048 Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments Status RECRUITING Phase N/A
NCT05761262 SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients Status UNKNOWN Phase N/A
NCT05760209 SMN Circular RNAs as Potential New Targets and Biomarkers for SMA Status UNKNOWN Phase N/A
NCT05712330 Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV Status COMPLETED Phase NA
NCT05663008 Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis Status RECRUITING Phase N/A
NCT05644899 Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study) Status COMPLETED Phase N/A
NCT05638750 Outpatient Rehabilitation Intervention for Young Children With SMA Status RECRUITING Phase NA
NCT05632666 Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA). Status UNKNOWN Phase NA
NCT05626855 Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Status ACTIVE_NOT_RECRUITING Phase PHASE3
NCT05539456 Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old Status UNKNOWN Phase N/A