Rare Genetic Diseases Resources
Patient Advocacy, Clinical Trials and Additional Information
At Aeglea we are dedicated to changing patients’ lives. Our credo is to put patients first. Successful research and drug development depends on patient advocacy and close collaboration with all members of a disease community including:
- Patients and their families and/or caregivers
- Physicians
- Metabolic dietitians
- Allied healthcare providers
- Advocacy organizations
- Research institutions
Currently, our patient advocacy programs focus on people affected by a rare metabolic diseases, including Arginase 1 Deficiency, Homocystinuria and Cystinuria. The Aeglea patient advocacy team connects with patient communities and organizations to ensure their voices are heard within our company and to better understand the needs of people living with these diseases.
We are conducting a clinical trial to assess the safety and efficacy of our enzyme replacement therapy in patients with Arginase 1 Deficiency.
For more information:
Find resources | Learn about clinical trials | Share your experiences
