30 closest matches · ranked by relevance
Fibrodysplasia Ossificans Progressiva
Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)
Fibrodysplasia Ossificans Progressiva
Primary: Parts A and B: Percentage of Flare-ups With No New Heterotopic Ossification (HO) at Week 12 — 64.3; 72.5 percentage of flare-ups
Fibrodysplasia Ossificans Progressiva
Primary: Percentage of Responders at Week 6 — 100; 88.9; 88.9 percentage of subjects — p=0.1664
Fibrodysplasia Ossificans Progressiva
Primary: Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs — 24; 20; 2; 4 Participants
Osteogenesis Imperfecta
Primary: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 — 2; 4; 2; 1 Participants
Fibrodysplasia Ossificans Progressiva (FOP)
Primary: Number of Participants With All Treatment-emergent Adverse Events (TEAEs), Serious and Non-serious Treatment-emergent Adverse Events and Serious and Non-serious…
Facioscapulohumeral Muscular Dystrophy
Primary: Bone Mineral Density in FSHD — -0.4 z-score
Fibrous Dysplasia Of Bone
Primary: Proportion of Participants With Serum Phosphate Levels Within the Target Range at Week 48 — 1 Proportion of participant
Familial Amyloid Polyneuropathy
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of participants
Osteogenesis Imperfecta, Type I · Osteogenesis Imperfecta Type III · Osteogenesis Imperfecta Type IV
Primary: Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 — 1.004; 0.993; 0.992 ratio — p=0.644
Osteogenesis Imperfecta
Primary: Spine Bone Mineral Density (BMD) — 6.1; 2.8 percentage of change in g/cm2
Diabetes Mellitus (DM)
Primary: Change in Metatarsal Phalangeal Joint Angle (Degrees) in People With Diabetes From Baseline and at a 3-year Period — 0.8; -0.1 degrees
Osteogenesis Imperfecta
Primary: Change in Bone Mineral Density in Response to Pamidronate — -3.54; -4.98 Z-score
Rothmund-Thomson Syndrome
Primary: Bone Density (Low Areal Bone Mineral Density (aBMD)) — -1.3; -0.9; -2.4; -1.2 z-score
Neurofibromatosis 1 · Neurofibroma, Plexiform
Primary: Median Time to Disease Progression — 13.2 Months
Adult Idiopathic Generalized Osteoporosis
Primary: Change in Lumbar Spine Bone Mineral Density (LS-BMD) on Active Medication — 10.1; 4.1 percentage of change
Idiopathic Pulmonary Fibrosis
Primary: Percentage of Participants With IPF Symptoms — 88.0; 51.8; 49.4; 42.9 Percentage of participants
Kidney Disease
Primary: Percentage Changes in Fibroblast Growth Factor-23 (FGF-23) Levels — -0.35; 0.25; 0.00; -0.02 percentage of change from baseline
Menopause · Fracture · Osteoporosis
Primary: Change in Lumbar Spine Bone Density by Dual Energy X-ray Absorptiometry (DXA) — 10.8 percentage of BMD change
Fibromyalgia
Primary: Health-related Quality of Life Assessed With the Revised Fibromyalgia Impact Questionnaire — 53.7; 55.5 score on a scale — p=0.740
Duchenne Muscular Dystrophy Cardiomyopathy · Cardiomyopathy, Dilated
Primary: Incidence of Treatment-Emergent Adverse Events — 14; 9; 10 Participants
Femoroacetabular Hip Impingement Syndrome
Primary: Prevalence of Femoroacetabular Impingement (FAI) — 28; 172 Participants
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Autosomal Dominant Osteopetrosis Type 2
Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Hypophosphatasia
Primary: Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) — -254.96; -564.27; 3.13; -397.72 ng/mL — p=0.0285
Lung Diseases, Interstitial
Primary: Incidence Probability of Progression to Pulmonary Fibrosing-Interstitial Lung Disease (PF-ILDs) — 0.147; 0.249; 0.334; 0.395 Cumulative incidence probability
Idiopathic Pulmonary Fibrosis
Primary: Idiopathic Pulmonary Fibrosis (IPF)-Related Costs — 38923.57; 22613.68; 20369.94; 37183.17 Euro (€) — p=0.0002
Hypophosphatasia
Primary: Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) — 2.00 scores on a scale
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
Osteoporosis · Atypical Femoral Fracture
Primary: Radiologic Evidence of Bone Healing — 12.6; 11.2; 15.4; 13.2 units on a scale