An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1/PHASE2
Clinical Trials Related to Spinal Muscular Atrophy
Related studies linked through entity extraction aliases.
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
- Status
- COMPLETED
- Phase
- N/A
Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment
- Status
- COMPLETED
- Phase
- NA
Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy
- Status
- COMPLETED
- Phase
- NA
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
- Status
- RECRUITING
- Phase
- N/A
Motor Function and Activity of Daily Life in Spinal Muscular Atrophy
- Status
- UNKNOWN
- Phase
- N/A
Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
- Status
- UNKNOWN
- Phase
- N/A
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
- Status
- RECRUITING
- Phase
- NA
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
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
- Status
- COMPLETED
- Phase
- PHASE2
Personalized Medicine for SMA: a Translational Project
- Status
- UNKNOWN
- Phase
- N/A
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
- Status
- RECRUITING
- Phase
- N/A
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
- Status
- RECRUITING
- Phase
- N/A
SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
- Status
- UNKNOWN
- Phase
- N/A
SMN Circular RNAs as Potential New Targets and Biomarkers for SMA
- Status
- UNKNOWN
- Phase
- N/A
Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
- Status
- COMPLETED
- Phase
- NA
Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis
- Status
- RECRUITING
- Phase
- N/A
Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)
- Status
- COMPLETED
- Phase
- N/A
Outpatient Rehabilitation Intervention for Young Children With SMA
- Status
- RECRUITING
- Phase
- NA
Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
- Status
- UNKNOWN
- Phase
- NA