Phase 2 N=29 Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
Progeria · Hutchinson-Gilford Syndrome
Primary: Proportion of Participants With Successful Rate of Weight Gain — 0.36 proportion of participants
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Phase 2 N=5 A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Progeria · Hutchinson-Gilford Syndrome
Primary: The Primary Objective of This Study is to Evaluate the Feasibility of Administering Intravenous Zoledronic Acid, Oral Pravastatin and Oral Lonafarnib, to Patients With…
N/A N=39 Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 2 N=22 Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
Short Stature
Primary: Height Standard Deviation Score — -2.52; -1.57; -1.19; -1.09 standard deviation score
Phase 2 N=7 IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
Short Stature
Primary: Height Velocity — 3.0; 4.3; 6.2; 4.8 cm/y
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale