BDSRA’s community was very active during this year’s Rare Disease Week.
Several hundred rare advocates visited their Congressional offices on Capitol Hill, February 27th and 28th. Among them were Jennifer VanHoutan from Illinois, and Sandra and Stephen Lehrman from North Carolina. Their children and grandchildren have been affected by CLN2 Batten disease.
This year the Rare Disease Legislative Advocates organized around these issues:
Orphan Products Extension Now, Accelerating Cures and Treatments (OPEN ACT) H.R.1223/S.1509
95% of rare disease have no FDA approved treatments. The OPEN ACT, bipartisan legislation supported by more than 270 patient organizations, could double the number of treatments available to rare disease patients by repurposing already approved drugs for rare diseases. If you or a loved one is on an off-label treatment, this legislation will be important to you.
Rare Disease Congressional Caucus
Advocates encouraged House and Senate members to become part of this bipartisan and bicameral Caucus to ensure that the rare disease community has a permanent voice on Capitol Hill.
On March 1st, BDSRA’s Executive Director Margie Frazier spoke at the National Institutes of Health Rare Disease Day celebration with a talk called: Collaborating for Successful Research. She and other panel members greeted several hundred attendees in person and several hundred more on livestream. The NIH Director, Francis Collins, opened the day-long celebration.
Advancing Access to Precision Medicine Act H.R.5062
On average, it takes 7 years for a rare disease patient to receive a diagnosis. This new legislation would provide coverage for genetic sequencing for patients to shorten the diagnostic odyssey.
Rare Disease Tree of Life Sculpture Dedication
Bekah, Danny and Ely Bowman from Orange County, CA traveled to BioMarin’s headquarters in San Rafael, to place beautiful ‘lockets of love’ with their boys’ names on them onto the tree sculpture. Bekah described the event on her Facebook feed:
“So neat to be at Biomarin for the unveiling of their “tree of life” sculpture. Locks were made with both Titus and Ely’s names (and it meant the world to us that they remembered our sweet Titus). The back of the locks are inscribed with “unlocking tomorrow’s together”. We were able to pick a special place on the tree of life where they will hang for always. Thank you for becoming family, Biomarin.”