Read the latest updates about BioMarin and learn more about our science, our people and the communities we support.
January 13, 2025
January 6, 2025
November 16, 2024
November 6, 2024
October 29, 2024
Today at the J.P. Morgan Healthcare Conference, our President and CEO Alexander Hardy and members of the BioMarin Leadership Team highlighted our continued progress against our strategy and how we are poised to deliver innovation for patients and families for decades to come.
Meet DMD. This gene provides instructions for making dystrophin, a protein that plays an essential role in strengthening and protecting muscle fibers. Changes in the DMD gene can result in a degenerative neuromuscular condition called Duchenne muscular dystrophy. We’re researching the potential to improve outcomes for people living with Duchenne muscular dystrophy by developing a medicine that may improve the levels of dystrophin in the muscle cells of those with the condition.
As we wrap up this year and look ahead to 2025, we are more driven than ever to deliver on our purpose: to be the biotech leader that translates the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The BioMarin 8 – our core Operating Principles – provide the framework for how we work together every day to fulfill that purpose. Thank you to all our employees for your hard work and dedication throughout 2024 – we look forward to what we will accomplish together in the new year.
We’re looking forward to the #TruePKU meeting next year, which will be the first opportunity for U.S. regulators to hear directly from the #PKU community in a public forum in more than a decade. We’re proud to support the National PKU Alliance's efforts to integrate the perspectives of people living with PKU into the process of developing new therapeutic options and are grateful that the #FDA has made this opportunity possible by granting this EL-PFDD.