Phase 2 N=35 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
Achondroplasia
Primary: Overall Summary of Adverse Events During Initial 6-Month Period — 8; 8; 10; 9 Participants
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Phase 2 N=75 A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Achondroplasia
Primary: Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs) — 11; 32; 32; 0 Participants
N/A N=248 A Study to Learn About Recifercept in Patients With Achondroplasia
Achondroplasia
Primary: Mean Height Growth at Month 3 — 1.11; 0.84; 0.75; 1.07 Ratio
Phase 2 N=57 A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
Achondroplasia
Primary: Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment — 4.09; 4.52; 5.16; 5.42 cm/year — p== 0.6004
Phase 3 N=121 A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Achondroplasia
Primary: Change From Baseline in Annualized Growth Velocity (AGV) at Week 52 — 1.71; 0.13 cm/year — p=< 0.0001
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=19 Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
Hypochondroplasia
Primary: Change From Baseline in Height-Standard Deviation Score (H-SDS) of Recombinant Human Growth Hormone (r-hGH) Treated Subjects at Year 3 — 1.39 Standard Deviation Score…
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