spinal-muscular-atrophy

Documents about spinal-muscular-atrophy

• Newborn Screening for Spinal Muscular Atrophy#981301ClinicalTrials.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
• Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy#1009382ClinicalTrials.gov
• Metabolomics of Children With SMA#804132ClinicalTrials.gov
• A Study of CK-2127107 in Patients With Spinal Muscular Atrophy#904193ClinicalTrials.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
• 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 Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients#899018ClinicalTrials.gov
• A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy#857984ClinicalTrials.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
• 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
• 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
• Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy#979951ClinicalTrials.gov
• Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments#831256ClinicalTrials.gov
• MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy#994813ClinicalTrials.gov
• Longitudinal Transcriptomic Analysis Reveals Systemic Effects of Risdiplam in Adults with Spinal Muscular Atrophy#314715Europe PMC
• Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"#899418ClinicalTrials.gov
• Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases#916900ClinicalTrials.gov
• Personalized Medicine for SMA: a Translational Project#825612ClinicalTrials.gov
• Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment#234973Europe PMC
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients#888824ClinicalTrials.gov
• Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies#858508ClinicalTrials.gov
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants#829182ClinicalTrials.gov
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy#913626ClinicalTrials.gov
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1#846225ClinicalTrials.gov
• Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy#884407ClinicalTrials.gov
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients#884448ClinicalTrials.gov
• Robot-assisted Training#870643ClinicalTrials.gov
• Real-world effectiveness and economic evaluations of EMA-approved orphan medicines for spinal muscular atrophy: a systematic literature review#200111ARPHA OAI-PMH Endpoint
• Skeletal muscle in spinal muscular atrophy: Critical insights from pathogenesis to therapeutic strategies#211218IRIS
• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia#918423ClinicalTrials.gov
• Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).#979624ClinicalTrials.gov
• Telerehabilitation in Proximal Muscle Weakness#870199ClinicalTrials.gov