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Mucopolysaccharidosis IV A
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IV A trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IV A trials you may qualify forThis trial aims to evaluate the prevalence of idiopathic short stature among children whose growth is above -2,5SD (AFPA- CRESS/Inserm -CompuGroup Medical 2018…
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, wit…
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH…
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and…
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
A Randomized, Double-Blind, Placebo-Controlled Direct-to-Consumer Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and…
The goal of this crossover clinical trial is to evaluate the efficacy and safety of Bloat Gummies on postprandial bloating and related gastrointestinal discomfo…