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Our Motivation | May 24, 2022

Inspired by the HAE Community, committed to helping make an impact

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. […]

Our Motivation | May 24, 2022

The unforgettable moments from “The Style Show: A Runway to Empower”

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.

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Our Motivation | April 29, 2022

The three lessons I’ve learned from planning a fashion show that are applicable to life

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.

Science | March 25, 2022

Advancing our understanding of gene therapy research, one publication at a time

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?

Science | February 4, 2022

Zeroing in on Therapeutic Drug Targets

Scientific pioneers Lon Cardon and Karol Estrada shed new light on the decades-old question of how to identify optimal drug targets.