Tags » National Human Genome Research Institute

NIH:Gene therapy shows promise for treating Niemann-Pick disease type C1

For the first time, National Institutes of Health researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). 497 more words


Tedx Talk: Dr. William Gahl

Dr. William Gahl who was called a “super–diagnostician” by the University of Wisconsin School of medicine, leads the Undiagnosed Diseases Program (UDP) at the NIH and essentially sees patients who lack a diagnosis after years of visits to other doctors. 170 more words

Chronic Illness

An outsider’s perspective on a medical data sharing conference

As a recent hire of Baylor College of Medicine Center for Medical Ethics and Health Policy, I was allowed a unique opportunity to watch high-level science policy discussion take place before I had much background knowledge on the issues. 373 more words