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Mucopolysaccharidosis IV A
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IV A trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IV A trials may be worth asking aboutNorth America
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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…
This study is a randomized, double-blind, placebo-controlled, within-individual crossover trial designed to assess the impact of regular use of a consumer-grade…
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establi…
This 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, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with A…
A Randomized, Double-Blind, Placebo-Controlled Direct-to-Consumer Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and…