Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
In a previous trial involving patients with early autosomal dominant polycystic kidney disease (ADPKD; estimated creatinine clearance, ≥60 ml per minute), the vasopressin V 2-receptor antagonist ...
Branchiootorenal spectrum disorder (BORSD) is a group of rare autosomal dominant conditions. It is characterized by malformations of the outer, middle, and inner ear associated with hearing impairment ...
Baylor College of Medicine offers preimplantation genetic testing, or PGT, to test embryos conceived through in vitro fertilization (IVF) for certain genetic conditions. PGT is an option available to ...