We're proud to announce that our Chief Commercial Officer Cristin Hubbard has been appointed to the Biotechnology Innovation Organization (BIO) Board of Directors. In her new role, Cristin will be a member of the organization’s Health Section Governing Board.

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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.

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RARE Scholars award recipient Brian DuVal is pursuing his Master of Public Health at Vanderbilt University in Tennessee. He aspires to become a leader in the hemophilia community and use his influence to improve the lives of others living with rare conditions.

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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.

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