Clinical Trial Search

Primary: Muscle Weakness — 2; 3 Participants

Primary: Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI) — 36.06; 88.47; 40.54; 89.44 scores on a scale

Primary: Achievement of Independent Sitting for at Least 30 Seconds — 13 Participants — p=<0.0001

Primary: Efficacy: Functional Motor Testing, Including Gross Motor Function Measure (GMFM) and Timed Motor Tests

Primary: Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical…

Primary: Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis — 0.208; -0.583 Overall Score — p=0.0083

Primary: Safety Labs

Primary: Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds — 14 Participants

Primary: Strength — 0.39 kilograms

Primary: Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] — -0.1; 0.0 score on a scale

Primary: Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group — 2.39; 0.51 Scores on…

Primary: Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=20kg — 5 milligram (mg)

Primary: Change From Baseline to Week 8 in Forced Vital Capacity (FVC) — -0.02; -0.03; -0.07 liters — p=0.9086

Primary: Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds — 14 Participants

Primary: Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds — 1 Participants

Primary: Safety: Frequency of Adverse Events/Lab Abnormalities

Primary: Laboratory Safety Data

Primary: Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) — 0; 0; 0; 0 Participants

Primary: Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA — 1; 8; 0; 1 Participants

Primary: Part 1: Selected Part 2 Dose of Risdiplam — 0.2 mg/kg

Primary: Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term) — 293.05; 299.50 meters

Primary: Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15 — -1.0; 3.9 scores on a scale — p=0.0000001

Primary: Time to Death or Respiratory Intervention — NA months

Primary: Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs — 8; 6; 9; 9 Participants

Primary: Muscle Strength Change From Baseline — -2.2; 3.1; -4.5; 1.3 percent change — p=0.28

Primary: Part B Infantile-onset SMA: Change From Baseline in CHOP-INTEND Total Score for 50/28mg Nusinersen Versus CS3B Matched Sham Control Group — 42.9; 16.9 score on scale…

Primary: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket — 7; 8; 9; 24 Participants

Primary: Scale for the Assessment and Rating of Ataxia(SARA) — 11.5; 9 score on a scale

Primary: Number of Participants With Adverse Events (AEs) — 12; 76; 70; 14 Participants

Primary: Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit — 25.0; 68.8 Percent of participants