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Genes
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Genes trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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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…
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approxi…
VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial…
This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's dis…
Background: PIEZO2 Deficiency Syndrome (PDS) is a genetic disorder that affects a person s ability to feel touches and pain. Researchers want to know more abou…
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to…
For Patients and Families Brief Title: Management of 1L Lorlatinib with Hyperlipidemia in ALK+ Advanced NSCLC What is this study about? This study is for peo…
Background: \- Genes are the instructions our body uses to function. Researchers can look for changes, or variants, in the genes. The goal of this study is to…