5,000 results on '"muscular atrophy, spinal"'
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2. A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (HINALEA 1)
3. A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
4. A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)
5. A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy (HINALEA 2)
6. A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma (ARISER)
7. A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NURTURE)
8. Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
9. Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam (STRENGTH)
10. Characterization of New Phenotypes of Patients with Spinal Muscular Atrophy Treated with SMN Restoring Therapy (PHENO SMART)
11. GCB-001 in Treatment of Patients with Type II (SMA) Spinal Muscular Atrophy (GITOPWTSMA)
12. A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) (PIERRE-PK)
13. A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy (Jewelfish)
14. Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS (CAPTURE ALS)
15. A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy (PUPFISH)
16. Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
17. Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
18. Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
19. Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (WeSMA)
20. Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy (PIERRE)
21. Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy (FANTASI-SMART)
22. Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
23. TRIAL READY (Clinical Trial Readiness)
24. Phenotype, Genotype & Biomarkers in ALS and Related Disorders
25. Clinical Procedures to Support Research in ALS (CAPTURE-ALS)
26. Phenotype, Genotype and Biomarkers 2 (PGB2)
27. Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA). (ACE SMA)
28. A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (Rainbowfish)
29. Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)
30. Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) (STEER)
31. Robot-assisted Training
32. Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) (NUSI-AD-5qSM)
33. An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (TOPAZ)
34. Spinraza in Adult Spinal Muscular Atrophy (SAS)
35. Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders (DANNIgene)
36. A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy (INFORM SMA)
37. Observational Study to Observe Variations of Gait Parameters in Patients with Neuromuscular Diseases
38. Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)
39. Two Different Treatment Modalities in Patients with Spinal Muscular Atrophy
40. Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
41. Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)
42. Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) (OFELIA)
43. An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
44. A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies (SHINE)
45. Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment
46. Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
47. Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
48. Wearable Technology to Assess Gait Function in SMA and DMD
49. Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
50. Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® (SMAII)
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