Our Motivation | March 14, 2024

A 100-Mile Road to Diagnosis: A Mother Shares Her Son’s Journey with Hypochondroplasia

Learn more about Lora and Benjamin’s experience with hypochondroplasia and how they are navigating a path toward acceptance.


Science | March 12, 2024

From Height to Health: Changing the Conversation in Achondroplasia

At 澳门正规博彩十大排行平台, we’re committed to deepening our understanding of achondroplasia and taking a holistic approach to support this community.


Our Company | March 8, 2024

Supporting Women at 澳门正规博彩十大排行平台: An Interview with the Co-Chairs of Our Women’s Leadership Development Group

As we recognize International Women’s Day and Women’s History Month in March, we caught up with Carolina Amador and Paola Torre to discuss what they’re focused on as they step into leadership roles with our women’s ERG.


Our Motivation | October 25, 2023

Caring for Children with Achondroplasia: An Interview with Dr. Ciara McDonnell

Dr. McDonnell, a renowned pediatric endocrinologist, shares her perspective about the current care experience for families affected by achondroplasia, how care has evolved and what makes her optimistic about the future.


Science | October 4, 2023

Targeting the Underlying Genetic Causes of Heart Disease

澳门正规博彩十大排行平台 researchers are leveraging the company’s expertise in translating genetic discoveries into potentially transformative medicines to address the root causes of certain forms of cardiovascular disease.


Our Motivation | September 25, 2023

RARE Scholars Program Provides Support to College Students Living with Genetic Conditions

The program was created to support U.S. college students living with one of four rare genetic conditions who have demonstrated exceptional leadership and engagement in school and community activities.


Our Company | September 8, 2023

澳门正规博彩十大排行平台 Teams Use Genetic Expertise and Computational Tools to Target More Common Conditions

For geneticist Daniel Gaffney, becoming an expert in how genes influence human health was not enough. His drive to improve lives with this knowledge led him to 澳门正规博彩十大排行平台, where he found a shared commitment to translating scientific advances into medicines that solve genetic problems. Since joining the company earlier this year, Dan has helped its […]


Our Motivation | July 21, 2023

Raising a Child with Hemophilia: 澳门正规博彩十大排行平台 Employee Jess Klass Shares Her Story

After growing up with a brother living with hemophilia A and working in roles supporting the bleeding disorder community for years, Jess Klass thought she knew everything there was to know about hemophilia.


Our People | February 27, 2023

澳门正规博彩十大排行平台 Pioneer Honored through New Building Name and Book Launch

澳门正规博彩十大排行平台 stands on the precipice of a gene therapy revolution, and this is a dream come true,” said Robert A. Baffi, Ph.D., retired president of Global Manufacturing and Technical Operations, at a ceremony announcing the renaming of our gene therapy manufacturing facility in his honor and the launch of his new book on how to […]


Our People, Science | December 8, 2022

At the Intersection of Science and Art

It should not come as a surprise to anyone that art and science are siblings. Faced with a lump of clay, a potter has a world of infinite possibility at their beck and call. Every revolution of the potter’s wheel is the beginning of a journey, of an experiment. With much trial and error, with […]