This review provides an updated summary of the state of our knowledge of the genetic contributions to the pathogenesis of congenital heart disease. Since 2007, when the initial American Heart ...

Odylia Therapeutics Inc. has announced it is working under a codevelopment partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by ...

"NPHP1-associated retinal dystrophy is an area where there is a huge opportunity to build on existing science and to develop an effective treatment, with an accelerated path to clinical trials. The ...