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Autosomal Recessive Disorder
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Autosomal Recessive Disorder trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Autosomal Recessive Disorder trials you may qualify forThis 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…
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 children who have hearin…
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free…
The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, i…
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…
The purpose of this study is to follow the natural history of non-syndromic hearing loss caused by mutations in two genes (GJB2 or OTOF) in children up to 16 ye…
This is a clinical intervention study of PKU carriers (cases) and non-carriers (controls). Upon completing the informed consent process, participants will compl…
This prospective observational study aims to investigate whether impulse oscillometry (IOS)-a non-invasive lung function test-can help predict the specific pres…
Background: \- Parkinson s disease is a disease of the nervous system that affects movement. People usually get it in their 70s or 80s. Early onset Parkinson s…