Clinical Trial Search

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: Safety Labs

Primary: Muscle Weakness — 2; 3 Participants

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

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

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

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

Primary: Laboratory Safety Data

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

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

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

Primary: Number of Participants With Treatment Emergent AEs and SAEs — 16; 11; 11; 3 Participants

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

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

Primary: Number of Participants With SABR Successfully Delivered in One Fraction — 30 Participants

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: Rate of Recruitment: Dyads — 2 dyads recruited and enrolled/week

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

Primary: Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) — 27.5; 91.6 percentage of participants

Primary: Strength — 0.39 kilograms

Primary: Change in 10 Meter Walk Test From Baseline in Gait Speed — 0.7051; 0.6606; 0.8544; 0.7547 meters per second

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

Primary: 12-month Primary Patency Rate — 143; 72 participants — p=0.437

Primary: Change in Six Minute Walk Test Between Device Turned ON and OFF - Distance Traveled — 837.1; 804.8 feet

Primary: The Proportion of MRI Scans From Pacemakers or CRT-Ps Providing Sufficient Image Quality to Allow for a Diagnostic Interpretation. — 129 MRI Scans

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

Primary: Carotid Intimal Media Thickness — -0.017; -0.092 mm

Primary: S-MRCP and S-EUS Concordance

Primary: Overview of Treatment-emergent Adverse Events by Age Subgroup — 27; 13; 4; 0 Participants