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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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Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being…
This is a non-randomized, one-arm, open label, single-center, phase I/II, prospective study, to assess safety, feasibility and efficacy of FT024 in 8 children (…
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim o…
This study will evaluate REGN5093 for the treatment of Non-Small Cell Lung Cancer (NSCLC) with MET alteration. The main purpose of this study is to determine th…
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and o…
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of…
This study will investigate why some people have Obstructive Sleep Apnea (OSA) and how the underlying cause may relate to OSA manifestations (including sleepine…
Background: Generalized Arterial Calcification of Infancy (GACI) is a very rare disorder. It can be fatal before birth or by age 6 months. Anumber of people wi…