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Below is information on current legislative actions that can impact the Barth syndrome community as well as the rare disease community at large. You can click on section headings to find more information about each bill. 

The STAT Act

The STAT Act, H.R. 1730/S. 670, is a bipartisan, bicameral, community-led bill aimed at improving the development of and access to therapies for the rare disease community. Learn more...


Better Empowerment Now to Enhance Framework and Improve Treatments Act of 2021 or the BENEFIT Act of 2021 requires the Food and Drug Administration (FDA) to consider relevant patient-focused drug development data, such as data from patient preference studies and patient-reported outcome data, in the risk-benefit assessment framework used in the process for approving new drugs.

After a new drug application has been approved, the FDA's public statement about how it used patient experience data shall include a description of how such data was considered in the risk-benefit assessment framework. Learn more...

Newborn Screening Saves Lives Reauthorization Act

This bill reauthorizes through FY2026 and revises several programs and activities relating to newborn screening for certain conditions and genetic, endocrine, and metabolic diseases.

Among its changes, the bill reauthorizes and makes mandatory

  • the Hunter Kelly Research Program at the National Institutes of Health, and
  • national surveillance activities conducted by the Centers for Disease Control and Prevention.

The bill also aligns statutory requirements for research on non-identified newborn blood spots with federal regulations governing research on human subjects. These blood spots are collected as part of newborn screening programs, and some states make them available for biomedical and public health research. Under current regulations, research on non-identified bio-specimens such as blood spots is not considered to be research on human subjects, to which additional protections apply. Learn more...

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