Clinical Trial Search

Primary: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement — 1; 0 participants

Primary: Change in FEV1 — -0.07; -0.08 Liters

Primary: Spouse Self-report of Resilience — 75.4; 75.9; 78.5; 81.0 units on a scale

Primary: Number of Hypoglycemic Events ≥50 mg/dl 15 Minutes AND ≥ 70 mg/dl 30 Minutes After Initial Treatment — 58; 53 Hypoglycemic Events — p=0.99

Primary: Schiff Air Test: Mean Reduction in Sensitivity Between Groups — -0.66; -1.12; -0.52; -1.11 units on a scale — p=0.05

Primary: Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24 — 0.08; -1.01 Ratio — p=< 0.0001

Primary: Change in Occupational Physical Activity From Baseline to 16 Weeks — 9752; -142 Average counts/work day

Primary: Engagement in Treatment - Treatment Completion (Number of Families Who Completed Treatment) — 27; 31 Families

Primary: Number of Participants With Any Adverse Event (AE), Serious Adverse Event (SAE) or Death During Part A — 3; 10; 0; 0 Participants

Primary: Relapse — 66; 99 Participants

Primary: Pain and Foreign Body Sensation — 89; 79; 83; 87 eyes

Primary: Number of Participants With Toxicity — 8 participants

Primary: Patients With Dose Limiting Toxicity — 0 Participants

Primary: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) — 14; 6; 9; 3 Participants

Primary: Self-report — 7.79; 6.49; 7.61; 7.73 units on a scale