NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
The following are issues that NAF is following. Click on the title of any issue to learn more or to take action to show your support.
This legislation would promote the development of drugs for pediatric cancers and rare diseases. It incentivizes the development of drugs to treat pediatric rare diseases through priority review vouchers (PRVs) and authorizes new studies on combinations of drugs that could be used to treat pediatric cancers. This legislation represents a huge opportunity for breakthroughs in treating kids who have rare diseases and cancer.
Currently, it can be very difficult to receive treatment from out-of-state doctors because of Medicaid enrollment requirements, although many rare disease patients must seek care from outof-state doctors as there are few specialists in their disease. This bill would make it simpler for doctors to enroll in Medicaid to treat out-of-state patients and would allow them to remain enrolled longer- making it easier for rare disease patients to receive the specialized care they need.
The Health Equity and Rare Disease Act of 2025 aims to increase research and investment in rare disease equity, specifically on conditions that affect minority populations. This would occur through focused research at the NIH, grant awards for this purpose, and specialized physician education and training programs.
The CDMRP was created to fill research gaps by funding high-impact, high-risk, and high-gain projects that other agencies may not venture to fund. This program was affected by a large funding cut in the FY25 budget continuing resolution. This initiative asks Congress to restore full funding for the CDMRP in FY26.
The Congresssionally Directed Medical Research Program’s (CDMRP) funding was reduced by 57% in FY25 from the FY24 level. Hereditary ataxia is aligible to receive funding through this program, but this decrease has lead to increased competition for a smaller total amount of money. This legislation proposes to restore CDMRP funding for FY25 to the FY24 level to continue funding biomedical research.
Medicaid provides coverage to millions of Americans- including children and those with disabilities. Congress is currently working on crafting the federal budget for FY26, which could include large cuts to Medicaid and make it harder for millions of people to receive healthcare. Urge Congress to preserve Medicaid for those who depend on it- including many in our community.
This legislation would make it easier for patients to access high costs treatments, like cell and gene therapies. This bill would encourage value-based purchasing arrangements for these treatments to ensure patients are paying for effective treatments.
This legislation encourages drug companies to invest in the development of treatments for rare diseases by increasing the orphan drug tax credit that is available to drug companies that develop these treatments.
This bill would work to lower patient copays by allowing amounts paid on behalf of the patient, by a nonprofit, drug company, or other entity, to count towards their copay- reducing the amount that they are ultimately responsible for paying.
Currently, people who receive supplemental security income (SSI) only maintain eligibility for it if they have less than $2,000 in assets, which does not allow these individuals to save money and create a safety net for themselves without losing these benefits. This threshold has not been updated since 1984. This legislation seeks to raise this number up to $10,000 for an individual and allows for increases on par with inflation.
This bill would incentivize international medical students to work in underserved or rural communities after graduation and increases the number of waivers that states can use for this purpose.
This bill allows taxpayers to receive a tax deduction for charitable donations even if they do not itemize their tax deductions- essentially making it easier to be rewarded for donating to charity and to incentivize people to donate.
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