Clinical Trial Search

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: Muscle Weakness — 2; 3 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

Primary: Mean TRS (Tremor Rating Score) — 26.6; 27.6 units on a scale

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: Safety Labs

Primary: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 13; 24; 65; 12 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: Safety: Frequency of Adverse Events/Lab Abnormalities

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

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

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

Primary: Strength — 0.39 kilograms

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

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