Tonight, I consider our journey one year ago and where my family sat one year ago, five years ago, seven years ago. I think of the connections made in Bethesda last year at The Inaugural International Conference of the Deficiency of ADA2, discovering other families who face the same journey.
I remember each of you. I remember your stories. I hope that you continue to speak out to raise awareness and help in our fight against orphan diseases, dada2, and PAN. Here are two stories that were shared during that time.
Please join us on November 24, 2017 for our 2nd annual Gala. Tickets can be purchased here.
Rare is defined as marked by unusual quality, merit, or appeal. We live through “rare” daily with Kelsey, some markedly easier than others. Sometimes it is the familiar that can help our “rare” become more usual. Today can be that day.
The EveryLife Foundation advocates for the rare disease community in Washington, D.C. A proposed bill, The OPEN ACT, that would help double the number of affordable treatments available to rare disease patients. This is a bipartisan bill that is supported by over 220 patient organizations. If put into law, it could bring hundreds of safe, effective, and affordable medicines to rare disease patients like Kelsey within the next several years. How? It provides incentives for drug creators to re-purpose therapies to help treat patients with life-threatening rare diseases. You can read For Rare Disease Patients: A New Pathway To Hundreds of New Therapies, in Health Affairs to learn more about The Open Act and the hundreds of new treatments it could provide for patients like Kelsey.
I was lucky enough to hear from Nancy Goodman, leading pediatric cancer advocate, founder, and executive director of Kids v Cancer, at the DADA2 Conference this past November. During her presentation, Ms. Goodman stated, “if there is a bill on Capitol Hill that will help your rare disease, then you should advocate for it!”
This is one of those times. If you feel inclined, please help our cause and thousands of others like us. You can click on this link to send a letter to your representative. It only takes about two minutes to complete the form.
SAVE THE DATE – DESIGNER BAG BINGO- JULY 13, 2017
St. Charles Borromeo * 176 Stagecoach Rd, Sicklerville, NJ 08081