REDACTED – FDA Family-Led Listening Session Report
On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with officials from the Food and Drug Administration (FDA). The session enabled those with ARG1-D and their caregivers to share their experiences, perspectives, needs, goals with officials from across the agency.
During the session the patients and caregivers shared:
- Difficulties obtaining a correct diagnosis
- Symptoms and experiences related to living with ARG1-D
- The impact or the condition on daily functioning and quality of life
- Experiences and challenges with existing treatments and the current state of disease management
- The need for disease awareness, improved care guidelines, and better treatment options