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235
datasets available to search
ShareScore release 0.9.0
Dataset results
235 results for “spinal muscular atrophy”
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
ClinicalTrials.gov study NCT00374075. IPD Sharing: Not stated. Countries: 1. Publications: 40.
Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy
ClinicalTrials.gov study NCT01369901. IPD Sharing: Not stated. Countries: 1. Publications: 3.
Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
ClinicalTrials.gov study NCT06599606. IPD Sharing: NO. Countries: 1. Publications: 15.
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-C
ClinicalTrials.gov study NCT02052791. IPD Sharing: Not stated. Countries: 1. Publications: 2.
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
ClinicalTrials.gov study NCT02391831. IPD Sharing: Not stated. Countries: 3. Publications: 1.
Mechanisms of Cell Death in Spinal Muscular Atrophy
ClinicalTrials.gov study NCT01754441. IPD Sharing: UNDECIDED. Countries: 1. Publications: 2.
Prospective Evaluation of Infants With Spinal Muscular Atrophy:
ClinicalTrials.gov study NCT02831296. IPD Sharing: YES. Countries: 1. Publications: 22.
Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
ClinicalTrials.gov study NCT03554343. IPD Sharing: NO. Countries: 1. Publications: 1.
Newborn Screening for Spinal Muscular Atrophy
ClinicalTrials.gov study NCT02123186. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
ClinicalTrials.gov study NCT05632666. IPD Sharing: NO. Countries: 1. Publications: 24.
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
ClinicalTrials.gov study NCT05522361. IPD Sharing: NO. Countries: 1. Publications: 3.
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
ClinicalTrials.gov study NCT05614531. IPD Sharing: NO. Countries: 1. Publications: 1.
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
ClinicalTrials.gov study NCT05878418. IPD Sharing: NO. Countries: 1. Publications: 1.
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
ClinicalTrials.gov study NCT01780246. IPD Sharing: Not stated. Countries: 1. Publications: 2.
Adult Spinal Muscular Atrophy (SMA) China Registry
ClinicalTrials.gov study NCT05618379. IPD Sharing: YES. Countries: 1. Publications: 1.
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
ClinicalTrials.gov study NCT05475691. IPD Sharing: Not stated. Countries: 6. Publications: 1.
Supplementary figures for: Respiratory trajectories in type 2 and 3 Spinal muscular atrophy in the iSMAC cohort study
<p>Objective. To describe the respiratory trajectories and their correlation with motor function in an international paediatric cohort of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA).</p> <p>Methods. Eight-year retrospective observational study of patients in the iSMAc natural history study. We retrieved anthropometrics, forced vital capacity (FVC) absolute, FVC% predicted (FVC%P.), Non-Invasive ventilation (NIV) requirement. Hammersmith functional motor scale (HFMS) and Revised performance of upper limb (RULM) were correlated with respiratory function. We excluded patients in interventional clinical trials and on Nusinersen commercial therapy.</p> <p>Results. There were 437 patients with SMA: 348 type 2, 89 non-ambulant type 3. Mean age at first visit was 6.9(±4.4) and 11.1(±4) years. In SMA type 2 FVC%P declined by 4.2%/year from 5 to 13 years, followed by a slower decline (1.0%/year). In type 3 FVC%P declined by 6.3%/year between 8 and 13 years, followed by a slower decline (0.9%/year). 39% SMA type 2 and 9% type 3 required NIV at median age 5.0(1.8-16.6) and 15.1(13.8-16.3) years. 84% SMA type 2 and 80% type 3 had scoliosis, 54% and 46% required surgery, which did not significantly affect respiratory decline. FVC%P positively correlated with HFMS and RULM in both subtypes.</p> <p>Conclusions. In SMA type 2 and non-ambulant type 3 lung function declines differently, with a common levelling after age 13 years. Lung and motor function correlated in both subtypes. Our data further defines the milder SMA phenotypes and provides novel information to benchmark the long-term efficacy of new treatments for SMA.</p>
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
ClinicalTrials.gov study NCT05861986. IPD Sharing: NO. Countries: 4. Publications: 0.
Hippotherapy in Children With Spinal Muscular Atrophy
ClinicalTrials.gov study NCT05341453. IPD Sharing: YES. Countries: 1. Publications: 0.
Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
ClinicalTrials.gov study NCT04262570. IPD Sharing: YES. Countries: 1. Publications: 0.
ScienceDex guides
Understand access before you commit
These curated guides explain access requirements, typical timelines, costs, and reuse considerations for widely used research datasets.
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DANDI Archive for NWB datasets
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International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.