spinal-muscular-atrophy

Documents about spinal-muscular-atrophy

• Newborn Screening for Spinal Muscular Atrophy#981301ClinicalTrials.gov
• Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam#1438175ClinicalTrials.gov
• New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy#907468ClinicalTrials.gov
• Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).#272379ClinicalTrials.gov
• Spinal Muscular Atrophy Neonatal Screening Program#876180ClinicalTrials.gov
• Clinical Study of Spinal Muscular Atrophy#1323402ClinicalTrials.gov
• Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy#1307564ClinicalTrials.gov
• Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy#1094988ClinicalTrials.gov
• Effect of Nusinersen on Adults With Spinal Muscular Atrophy#1444713ClinicalTrials.gov
• Body Weight Support Harness System in Spinal Muscular Atrophy#1446444ClinicalTrials.gov
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III#1320358ClinicalTrials.gov
• Palliative Care in Spinal Muscular Atrophy (SMA) 1#1046445ClinicalTrials.gov
• Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development#1125148ClinicalTrials.gov
• Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy#1395125ClinicalTrials.gov
• VRehab-SMA Phase 1.2#1465204ClinicalTrials.gov
• Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy#1009382ClinicalTrials.gov
• Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy#1396308ClinicalTrials.gov
• Metabolomics of Children With SMA#804132ClinicalTrials.gov
• A Study of CK-2127107 in Patients With Spinal Muscular Atrophy#904193ClinicalTrials.gov
• Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)#1095276ClinicalTrials.gov
• A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy#880564ClinicalTrials.gov
• Natural History of SMA#866283ClinicalTrials.gov
• Motor Function and Activity of Daily Life in Spinal Muscular Atrophy#905613ClinicalTrials.gov
• Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women#1280188ClinicalTrials.gov
• CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I#873227ClinicalTrials.gov
• Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy#893851ClinicalTrials.gov
• Mechanisms of Cell Death in Spinal Muscular Atrophy#803423ClinicalTrials.gov
• A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy#1464510ClinicalTrials.gov
• A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients#899018ClinicalTrials.gov
• Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy#1111421ClinicalTrials.gov
• A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy#857984ClinicalTrials.gov
• Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions#1261158ClinicalTrials.gov
• Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy#1376359ClinicalTrials.gov
• UK SMA Patient Registry#993740ClinicalTrials.gov
• A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy#981996ClinicalTrials.gov
• A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis#982687ClinicalTrials.gov
• High-intensity Interval Training in Patients With Spinal Muscular Atrophy#1171155ClinicalTrials.gov
• European Registry of Patients With Infantile-onset Spinal Muscular Atrophy#1309216ClinicalTrials.gov
• Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)#803785ClinicalTrials.gov
• Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)#852465ClinicalTrials.gov
• Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen#859209ClinicalTrials.gov
• Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy#878351ClinicalTrials.gov
• A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients#836982ClinicalTrials.gov
• A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy#910266ClinicalTrials.gov
• A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy#1115118ClinicalTrials.gov
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I#802789ClinicalTrials.gov
• Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies#831208ClinicalTrials.gov
• An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)#857012ClinicalTrials.gov
• Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients#810876ClinicalTrials.gov
• Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy#840648ClinicalTrials.gov