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.
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.
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.
Read More read more ⌃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 […]
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.
澳门十大博彩正规游戏平台 Approves 澳门正规博彩十大排行平台’s Gene Therapy for a Genetic Bleeding Disorder
We’re thrilled to share that the U.S. Food and Drug Administration has approved our gene therapy for a genetic bleeding disorder. Developing the one-time therapy and bringing it over the finish line, first in Europe and now the United States, has been a global effort. Our teams have made unprecedented scientific, medical, manufacturing and regulatory […]
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 ⌃