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Clinical trial condition

Mucopolysaccharidosis III

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

41 public trials 8 recruiting studies
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Mucopolysaccharidosis III trial results

Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
  • Women's and Children's Hospital North Adelaide, South Australia, Australia
  • Vall d'Hebron Barcelona Hospital Campus Barcelona, Spain
View Trial Details
Recruiting

Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance, California, United States
View Trial Details
Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

NCT06036693

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
Locations
  • Centre Hospitalier Universitaire d'Angers Angers, France
  • Hôpital des Enfants - Groupe Hospitalier Pellegrin Bordeaux, France
  • Hôpital Morvan Brest, France
  • Hôpital d'Estaing Clermont-Ferrand, France
View all 23 locations
View Trial Details
Recruiting

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • National Center for Child Health and Development Setagaya City, Tokyo, Japan
  • Ajou University Medical Center Suwon, Gyeongi-do, South Korea
View Trial Details
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
View Trial Details
Enrolling by Invitation

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • Women's and Children's Hospital North Adelaide, South Australia, Australia
  • Vall d'Hebron Barcelona Campus Barcelona, Spain
  • Hospital Clínico Universitario de Santiago Santiago de Compostela, Spain
View Trial Details

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