10 closest matches · ranked by relevance
15q Duplication Syndrome · CDKL5 Deficiency Disease
Primary: Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period — 11.7; -23.6 percent change
22q11 Deletion Syndrome
Primary: Safety and Tolerability of NB-001 — 18; 24; 14; 20 Participants
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
22q13 Deletion Syndrome · Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1 — 15.78; 11.22; 7.61; 9.70 score on a scale
Duchenne Muscular Dystrophy
Primary: Number of Participants With Unacceptable Toxicity. — 0 Participants
22Q11 Deletion Syndrome
Primary: Change From Baseline in Adolescents' Coping Skills — 3.10; 2.68; 3.18; 2.79 units on a scale — p=0.04
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Fragile X Syndrome
Primary: Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients…
Angelman Syndrome · Nervous System Diseases
Primary: Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills — 5.7; 4.7; 5.8…
Down Syndrome · Obstructive Sleep Apnea Syndrome
Primary: Change in Epworth Sleepiness Scale From Baseline to End of Study — -1; -0.5; 1 units on a scale