When someone is fighting a fatal or debilitating disease for which no drugs are available, every day counts. We apply the right science and the right development strategies to rapidly develop treatments for people who urgently need them.
Galactosemia
OUR COMMITMENT TO THE GALACTOSEMIA COMMUNITY
Learn more about Galactosemia at Galactosemia Together
Patient Advocacy Organizations
- Galactosemia Foundation
The Galactosemia Foundation is a nonprofit charitable organization that advocates for people with Galactosemia and their families.
SORD DEFICIENCY
OUR COMMITMENT TO THE SORD DEFICIENCY COMMUNITY
Patient Advocacy Organizations
- Charcot-Marie-Tooth Association (CMTA)
CMTA is devoted to supporting the development of new drugs to treat CMT, improving the quality of life for people with CMT, and, ultimately, finding a cure.
- Hereditary Neuropathy Foundation (HNF)
HNF is a nonprofit organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth (CMT) and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures.
- CMT Research Foundation (CMTRF)
The CMT Research Foundation is a patient-led, non-profit with one single mission: to raise funds to invest in science that will lead to treatments and cures for Charcot-Marie-Tooth disease.
PMM2-CDG
OUR COMMITMENT TO THE PMM2-CDG COMMUNITY
Patient Advocacy Organizations
- CDG CARE
The mission of CDG CARE is to promote greater awareness and understanding of CDG, to provide information and support to families affected by CDG, and to advocate for and fund scientific research to advance the diagnosis and treatment of CDG.
In the patients & caregivers section
