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Conditions: Muscular Dystrophy, Muscular Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 5 Years to 17 Years · Male only
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions: Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.; Diagnostic Test
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Wilmington, Delaware

Conditions: Pompe Disease
Interventions: Pyridostigmine Bromide; Drug
Lead sponsor: University of Florida; Other
Eligibility: 8 Years to 60 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Achondroplasia
Interventions: BMN 111, Placebo; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Up to 59 Months
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 9
States / cities: Oakland, California • Torrance, California • Wilmington, Delaware + 6 more

Conditions: Fibrodysplasia Ossificans Progressiva (FOP)
Interventions: Non-Interventional; Other
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: Not listed
Enrollment: 131 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023
U.S. locations: 1
States / cities: Tarrytown, New York

Conditions: Danon Disease
Interventions: RP-A501; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 3
States / cities: La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania

Conditions: Cardiomyopathy, Hypertrophic, Muscular Dystrophy, Oculopharyngeal
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
Interventions: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:; Drug
Lead sponsor: Daniel Benjamin; Other
Eligibility: Up to 21 Years
Enrollment: 3,520 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2019
U.S. locations: 43
States / cities: Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more

Conditions: Hypochondroplasia
Interventions: Vosoritide, Placebo; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 3 Years to 17 Years
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 4
States / cities: Wilmington, Delaware • Washington D.C., District of Columbia • Chicago, Illinois + 1 more

Conditions: Becker Muscular Dystrophy
Interventions: (-)-Epicatechin; Drug
Lead sponsor: Craig McDonald, MD; Other
Eligibility: 18 Years to 70 Years · Male only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Skeletal Disorders
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 2 Months to 100 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Structural Anomalies, Cardiac Anomalies, Central Nervous System Anomalies, Thorax Anomalies, Genito-urinary Anomalies, Gastrointestinal Anomalies, Skeletal Anomalies, Multiple Anomalies
Interventions: Whole Exome Sequencing (WES); Device
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 64 Years · Female only
Enrollment: 316 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Enchondromatosis
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 2 Years to 100 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Becker Muscular Dystrophy
Interventions: (-)-epicatechin; Drug
Lead sponsor: Craig McDonald, MD; Other
Eligibility: 18 Years to 60 Years · Male only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions: Cerezyme (imiglucerase for injection); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 10 Years to 65 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2004
U.S. locations: 1
States / cities: Coral Springs, Florida

Conditions: Hypophosphatemia, Familial, Hyperparathyroidism
Interventions: Paricalcitol, Placebo; Drug · Other
Lead sponsor: Yale University; Other
Eligibility: 9 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Achondroplasia
Interventions: Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration; Drug
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 0 Years to 32 Months
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 3
States / cities: Oakland, California • Baltimore, Maryland • Madison, Wisconsin

Conditions: Hypochondroplasia
Interventions: Infigratinib; Drug
Lead sponsor: QED Therapeutics, a BridgeBio company; Industry
Eligibility: 3 Years to 18 Years
Enrollment: 135 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2036
U.S. locations: 8
States / cities: Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more

Conditions: Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions: Dried blood spot test for MPS; Other
Lead sponsor: National MPS Society; Other
Eligibility: 6 Months to 18 Years
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 2
States / cities: New Brunswick, New Jersey • New York, New York

Conditions: Musculoskeletal Abnormalities, Obesity, Sarcopenic Obesity, Osteoporosis, Drug Effect
Interventions: Tirzepatide, Lifestyle toolkit; Drug · Behavioral
Lead sponsor: Pennington Biomedical Research Center; Other
Eligibility: 18 Years to 50 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Baton Rouge, Louisiana

Conditions: Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Interventions: Not listed
Lead sponsor: Global Healthy Living Foundation; Other
Eligibility: 19 Years and older
Enrollment: 40,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2025
U.S. locations: 1
States / cities: Upper Nyack, New York

Conditions: Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Interventions: Burosumab; Drug
Lead sponsor: Laura Tosi; Other
Eligibility: Not listed
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Osteogenesis Imperfecta
Interventions: Risedronate Oral Tablet; Drug
Lead sponsor: Hospital for Special Surgery, New York; Other
Eligibility: 6 Years to 100 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: New York, New York

Conditions: Cystic Fibrosis
Interventions: Magnetic resonance relaxometry, Magnetic resonance spectroscopy, Blood Draw, DXA, pQCT; Diagnostic Test
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 13 Years to 20 Years
Enrollment: 36 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Boston, Massachusetts

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Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

Non-invasive Functional Assessment and Pathogenesis of Morquio A

A Pilot Study of Pyridostigmine in Pompe Disease

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Study of Muscle Abnormalities in Patients With Specific Genetic Mutations

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

(-)- Epicatechin Becker Muscular Dystrophy

Study of Skeletal Disorders

Clinical Utility of Prenatal Whole Exome Sequencing

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

GLP-1R Actions on Muscle and the Skeleton

PatientSpot Formerly Known as ArthritisPower

Burosumab for CSHS

Effects of Bisphosphonates on OI-Related Hearing Loss

Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis