30 closest matches · ranked by relevance
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…
Angelman Syndrome
Primary: Bayley Scales of Infant and Toddler Development, 2nd Edition (BSID-II) Score at Baseline, 8 Weeks After Treatment, and at 16 Week Follow-up — 26.4; 31.7; 30.7; 18.9…
Angelman Syndrome
Primary: Bayley Cognitive Age Equivalent at 1 Year — 19; 19 months — p=0.75
Angelman Syndrome
Primary: Maximum Dose of Levodopa/Carbidopa That Can be Tolerated (Without Any Dose Limiting Toxicity) by at Least 3 Subjects. — 15 mg/kg/day
Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
Primary: Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks — 1; 2; 11; 11 Participants
Angelman Syndrome
Primary: Incidence of Adverse Events in Placebo and Active Treatment Groups — 25; 27; 25; 23 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
Fragile X Syndrome · Autism Spectrum Disorders
Primary: Clinical Global Impression- Severity Scale (CGI-S) — 7.0 units on a scale
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
Fragile X Syndrome
Primary: Contingency Naming Test (CNT) Performance Score — 37.05; 40.50; 66.94; 61.56 correct responses per minute
Photosensitive Epilepsy
Primary: % Change in Frequency of Handwaving Episodes — -66.2 % change in hand waving episodes — p=<0.001
Smith-Lemli-Opitz Syndrome
Primary: Hyperactivity Sub-scale of the Aberrant Behavior Checklist-Community (ABC-C). — 8.6; 8.6 units on a scale — p=1.0
Phelan-McDermid Syndrome
Primary: Safety and Tolerability — 17; 17; 1; 3 Participants
Fragile x Syndrome
Primary: Clinical Global Impression-Improvement (CGI-I) Scale — 3.4; 3.5 Scores on a scale — p=0.4475
Down Syndrome
Primary: Step Width — 10.4 cm
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Smith-Lemli-Opitz Syndrome
Primary: Change in Plasma Cholesterol — 37.8 change (%) compared to baseline — p=0.011
Sleep Apnea, Obstructive · Down Syndrome
Primary: Serious Adverse Events — 7 participants
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
Fragile X Syndrome
Primary: Change in Weighted Child Intentional Communication Score — 0.14; 0.04 log10(scores on a scale) — p=0.1587
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Klinefelter Syndrome
Primary: Motor Function/Strength — 86; 81; 92; 89 score on a scale — p=0.005
22q11 Deletion Syndrome
Primary: Safety and Tolerability of NB-001 — 18; 24; 14; 20 Participants
Down Syndrome
Primary: Incidence of Adverse Events (TEAEs) — 5; 2; 0 participants
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…
Fragile X Syndrome
Primary: Change in Mullen Scales of Early Learning - Expressive Language Raw Score — 19.3; 21.3; 21.3; 25.04 units on a scale
Fragile X Syndrome
Primary: Aberrant Behavior Checklist — 7.1; 15.6; 9.6; 7.6 units on a scale
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events