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Mucopolysaccharidoses

Clinical trial condition

Mucopolysaccharidoses

Explore clinical trials studying Mucopolysaccharidoses. Study availability and eligibility vary by location and protocol.

193 public trials 26 recruiting studies

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Mucopolysaccharidoses trial results

Recruiting

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

NCT06488924

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Hiroshima University Hospital Hiroshima, Japan

University of the Ryukyus Hospital Okinawa, Japan

National Center for Child Health and Development Tokyo, Japan

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Recruiting

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

NCT06614894

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations: Lysosomal & Rare Disorders Research & Treatment Center, Inc. Fairfax, Virginia, United States

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Recruiting

Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation

NCT06551961

Conditions: Carbohydrate Metabolism, Inborn Errors, Colonic Diseases, Colonic Diseases, Functional, +15 more
Locations: Nottingham Digestive Disease Centre Nottingham, Nottinghamshire, United Kingdom

Nottingham Digestive Diseases Centre Nottingham, Please Choose..., United Kingdom

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Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I

NCT06519552

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: West China Hospital, Sichuan Universit Chengdu, Sichuan, China

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Recruiting

A Natural History Study in Children With a Type II Collagen Disorder With Short Stature

NCT05408715

Conditions: Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +20 more
Locations: Hopital Necker-Enfants Malades Paris, France

Hospital Universitario La Paz Madrid, Spain

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Recruiting

Decoding the Genetic Landscape of Skeletal Diseases

NCT05876416

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations: Karolinska University Hospital Stockholm, Sweden

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Not yet recruiting

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

NCT07579910

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Not specified

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Not yet recruiting

Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

NCT07516587

Conditions: Abdominal Discomfort, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations: Alethios, Inc. San Francisco, California, United States

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Enrolling by invitation

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

NCT07393373

Conditions: Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +16 more
Locations: UCSF Benioff Children's Hospital Oakland, California, United States

Childrens Hospital Colorado Aurora, Colorado, United States

Children's National Hospital Washington D.C., District of Columbia, United States

Johns Hopkins School of Medicine Baltimore, Maryland, United States

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Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038

Conditions: Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
Locations: Maimonides Medical Center Brooklyn, New York, United States

NYU Langone Hospital - Brooklyn Brooklyn, New York, United States

North Shore University Hospital Manhasset, New York, United States

NYU Langone Health - Tisch Hospital New York, United States

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Mucopolysaccharidoses

Mucopolysaccharidoses trial results

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