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Mucopolysaccharidosis IV clinical trials recruiting in the U.S.

11 studies for Mucopolysaccharidosis IV are recruiting participants at sites across the United States.

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Recently updated studies

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

RecruitingObservational study
Ages 2 to 100 · All sexes · Updated Oct 1, 2026 · NCT06749366

Study of Skeletal Disorders

RecruitingObservational studyHealthy volunteers welcome
2 Months – 100 Years · All sexes · Updated Jul 20, 2026 · NCT05031507

Prospective Clinical Assessment Study in Children With Hypochondroplasia

RecruitingObservational study
30 Months – 16 Years · All sexes · Updated Jul 16, 2026 · NCT06410976

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

RecruitingPhase 2
0 Years – 32 Months · All sexes · Updated Jul 13, 2026 · NCT07169279

A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating

RecruitingHealthy volunteers welcome
Ages 18 and older · All sexes · Updated May 15, 2026 · NCT07591584

Non-invasive Functional Assessment and Pathogenesis of Morquio A

RecruitingObservational study
All ages · All sexes · Updated May 13, 2026 · NCT05284006

Effects of Bisphosphonates on OI-Related Hearing Loss

RecruitingPhase 4Healthy volunteers welcome
Ages 6 to 100 · All sexes · Updated Apr 28, 2026 · NCT04152551

Registry of Patients Diagnosed With Lysosomal Storage Diseases

RecruitingObservational study
Up to age 64 · All sexes · Updated Apr 8, 2026 · NCT05619900

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

RecruitingPhase 1
Ages 18 to 50 · Women · Updated Mar 17, 2026 · NCT04532047

Longitudinal Study of Neurodegenerative Disorders

RecruitingObservational study
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200

Cardiac Structure and Function in MPS

RecruitingObservational studyHealthy volunteers welcome
Ages 0 to 99 · All sexes · Updated Jan 26, 2026 · NCT07361536

From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.