Current trials in SMA
TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.
Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov – companies are obliged to provide details of their trials there.
The table below lists all SMA trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.
Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.
Trials for SMA listed on clinicaltrials.gov
- A Study of Spinraza (Nusinersen) Exposure in Pregnant Women With Spinal Muscular Atrophy (SMA) Within Existing SMA Registries
- Personalized Medicine for SMA: a Translational Project
- Impact of Covid-19 Pandemic on Children With Neuromuscular Disease and Their Parents in Turkey: Parent Perspective
- MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
- Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
- SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
- SMN Circular RNAs as Potential New Targets and Biomarkers for SMA
- Natural History of SMA
- Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
- Investigation of the Usability of the Dynamic Scaffolding System
- Body Weight Support Harness System in Spinal Muscular Atrophy
- Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
- Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis
- Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)
- Feasibility of a Newborn Screening for Spinal Muscular Atrophy (SMA) in France: DEPISMA Prefigurator Project in Grand-Est and Nouvelle-Aquitaine