Pennsylvania Senate OK’s bill to support rare disease community
Legislation would extend PARDAC's mandate to 2028
The Pennsylvania Senate has approved legislation to extend support for patients with rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency, by continuing the work of the Pennsylvania Rare Disease Advisory Council (PARDAC).
PARDAC was established in 2017 to help Pennsylvania residents navigate life with a rare disease by advancing research, raising awareness, and advising policymakers.
A bipartisan effort sponsored by Republican Sen. Judy Ward, Senate Bill 715 extends PARDAC’s mandate through 2028. Without the legislation, the council would have ended in 2025.
Rare diseases are defined as those that affect fewer than 200,000 people in the U.S. While each disease is uncommon, there are more than 7,000 known rare diseases and over 250 new ones are identified each year. In Pennsylvania alone, approximately 1.2 million residents are living with a rare disease.
AADC deficiency is among the rarest, with fewer than 150 diagnosed cases worldwide. A genetic disorder, the disease disrupts how nerve cells in the brain communicate, causing developmental delays and symptoms such as muscle stiffness, and weakness.
Like a number of rare diseases, AADC deficiency requires ongoing research and better public understanding. The first disease-modifying therapy for AADC deficiency in the U.S. was approved by the U.S. Food and Drug Administration in 2024. The therapy is also approved in the European Union and in the U.K., where it’s sold as Upstaza. Still, for many patients, the journey to an accurate diagnosis and proper care can take years, often involving multiple specialists and misdiagnoses.
Challenges of a rare disease
Ward, who has a background in nursing, said she understands the daily challenges faced by people with rare diseases.
“As a nurse, I know firsthand the impact a rare disease can have on someone and their loved ones and the daily challenges it can present,” Ward said in a press release from her office. “This community needs our help and that is why the work of the PA Rare Disease Advisory Council could not be more necessary. Senate Bill 715 will build on the efforts of the Council and enable them to continue their amazing work.”
PARDAC plays a key role in advising state leaders and shaping policies. The council gathers data, engages with patients, and works with medical experts to recommend solutions that improve care and access.
Senate Bill 715 received unanimous bipartisan support in the Senate and now moves to the state’s House of Representatives for further consideration. Ward will provide updates about the bill and other legislative initiatives on Facebook, X/Twitter, or on her Senate website.
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