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.
It’s time to give a platform, or in my case “runway,” to people with rare disease
In a special guest series, Kendra Gottsleben takes us on her journey of planning a fashion show to celebrate diversity in all its forms.
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?
Read More read more ⌃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.
BioMarin’s Center of Technology empowers scientists with technology, resources, and collaboration
For a scientist, access to cutting-edge technologies, rare resources, and expert support can turn a new idea into a groundbreaking discovery.
Meet the Scientist: Christian Vettermann, Ph.D.
In his latest paper, Dr. Vettermann solves a complex problem facing hemophilia A scientists and clinicians for years.
Clinical Trials
Our thoughtful approach to clinical trial design enables us to move fast and efficiently while protecting the safety of our patients.
Learn More about our clinical trials ⌃