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Inherited Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Disease 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.
This is a single site, single arm, non-randomized, pilot trial to assess the feasibility and safety of treating severe congenital diaphragmatic hernia (CDH) wit…
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown th…
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficienc…
Next generation sequencing (NGS) allows some better diagnostic results, particularly, in the rare diseases field. At a twenty five percent rate, those exams hig…
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).
The purpose of the study is to evaluate the maternal and fetal outcomes of a new technique for the fetoscopic repair of fetal MMC at Texas Children's Hospital P…
The primary objective of this protocol is to evaluate the response rate of bilateral disease participants who have at least one eye with advanced intra-ocular r…