Last week on HAE Day, we joined the community and HAEi in the effort to raise awareness of HAE. The first report of the hereditary form of angioedema was published almost 140 years ago by Sir William Osler. It took 75 years, until 1963, for the biochemical basis of Hereditary Angioedema (HAE) to be elucidated. […]
I am still finding it hard to believe that my lifelong dream of the “The Style Show: A Runway to Empower” is now finally a reality. In front of a sold-out room, 17 models of all shapes, sizes and abilities strutted down the runway representing true diversity in all its forms. I can still feel […]
Independent Research Presented at Pediatric Endocrine Society Annual Meeting
BioMarin congratulates Dr. Andrew Dauber, Chief of Endocrinology at Children’s National Hospital in Washington, D.C., who has recently presented independent research to study a BioMarin therapy, in selected genetic causes of short stature.Read More read more ⌃
There are three lessons that I’ve learned along the way that I think are applicable to any life situation, so I wanted to pass them along to all of you.
Forty years ago, the idea of gene therapy seemed like science fiction. Deliver a genetic blueprint to human cells using modified viruses or other molecular tools?