Patients Rising is trying support the Promising Pathways Act, a bipartisan bill introduced by Senators Braun(R), Gillibrand(D), and Wicker(R). To do this, we are asking for stories from the rare, and ultra-rare disease community to give perspective to the importance of the Act. Senators Bruan and Gillibrand are organizing a media event on Oct. 26 in support of the bi-partisan bill. We are helping the Senators collect videos and written testimony from the patient and caregiver community.

For individuals with life-threatening, serious diseases, timely access to treatment is an essential element in the fight for life. The Promising Pathway Act would grant patients with rare, progressive, and similar serious diseases earlier access to safe and promising therapies.

WHAT WE ARE ASKING YOU TO DO:

We want to know what have your experiences with rare and ultra-rare conditions have been

• How has it affected your life (good or bad)?
• Do you feel well-served by the mission or actions of the Food and Drug Administration? Please explain.
• Are you willing to use drugs for your condition that have not received full approval by the FDA?