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Autosomal Recessive Disorder
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Autosomal Recessive Disorder trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Autosomal Recessive Disorder trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired pri…
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult part…
A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Clinical Activity of ADX-097 Administered Subcutaneously in Male and Female Part…
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of a…
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations…
This study is a randomized, double-blind, multicenter, placebo-controlled Phase III clinical trial designed to evaluate the efficacy, safety, and Pop PK charact…
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 De…