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.
We are energized by this and yet are acutely aware of how much more there is to do for all forms of the disease. It truly takes a long-term commitment to support, advocate, and build a better future for children and families impacted by Batten disease.