Clinical Trial Search

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

Primary: Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability — 2; 4; 2; 1 Participants

Primary: Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] — 4.1402; 3.8580; -0.0871; -0.1042 cm³

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: Count of Adverse Events Reported — 109; 52 adverse events reported

Primary: Number of Participants With Adverse Events as a Measure of Safety and Tolerability — 0; 2; 10 Participants

Primary: Muscle Mass Via Dual-Energy X-ray Absortiometry (DEXA)Scan Data — 6104.45; 6355.3 grams — p=.318

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

Primary: Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA — 1; 8; 0; 1 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: Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds — 14 Participants

Primary: Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA) — -25.9; -27.8 percentage change — p=0.29

Primary: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths. — 48; 50; 44; 49 Participants

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

Primary: Strength — 0.39 kilograms

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

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: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52 — 8.63; 9.63; -10.27; -8.96 meters — p=0.2210

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

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

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

Primary: Change From Baseline Walking Time in 10 Meter Walk Test to the Measurement Taken Directly After rTMS — -5.41; 1.70 seconds

Primary: Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician — 3.7; 3.8 score on a scale

Primary: Safety Labs

Primary: Time to Death or Respiratory Intervention — NA months

Primary: Symptom Severity — 28.0; 18.6; 14.2; 12.3 score on a scale — p=0.01

Primary: Change From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756