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 SERPINA1. This gene provides instructions for making the alpha-1 antitrypsin (AAT) protein, which forms in the liver and protects tissues in the lungs from enzyme damage. Changes in the SERPINA1 gene can reduce the body’s supply of AAT and cause a genetic condition called alpha-1 antitrypsin deficiency (AATD), which impacts the function of the liver and/or lungs.

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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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It’s been exciting to connect with and learn from the community at the first Little Legs Big Heart Skeletal Dysplasia Conference this weekend in West Palm Beach, Florida. In addition to hearing from members of the community, we had the opportunity to share updates about our research in #achondroplasia and #hypochondroplasia. Thanks to Kristen DeAndrade and The Little Legs Big Heart Foundation for organizing this event and inviting us to be part of it.

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