Congressman Paul Tonko Visits UAlbany to Celebrate the HEART Act
By Erin Frick
ALBANY, N.Y. (Feb. 16, 2023) — Congressman Paul Tonko visited the RNA Institute on Tuesday for a press conference celebrating the passage of the Helping Experts Accelerate Rare Treatments (HEART) Act, which was signed into law in December.
The new legislation will strengthen the U.S. Food and Drug Administration (FDA) review process for drugs that treat rare and ultra-rare diseases by authorizing new studies in the fields of rare disease research, and directly involving scientists, doctors and patients with expertise throughout the review process.
“At UAlbany, we know all too well the challenges presented by studying rare diseases,” said UAlbany President Havidán Rodríguez. “There are over 7,000 rare and ultra-rare diseases which impact an estimated 25-30 million Americans. And yet, only 5% of these diseases have FDA-approved treatments. Rare diseases are often hampered by the inability to complete high-quality clinical trials given the small number of patients impacted by each disease.
“The research being conducted here at the University at Albany’s RNA Institute on rare neuromuscular and metabolic diseases is of critical significance, not only to those afflicted by the disease themselves, but also their families and loved ones. If not for the dedication and commitment of the scientists and researchers that work tirelessly to advance therapeutic approaches, millions of Americans would be left without hope. Congressman Tonko’s leadership in the areas of rare disease research, and support of the HEART Act, will ensure that patients living with these diseases will receive greater access to treatments, saving lives.”
Tonko was joined at the event by Capital Region community members affected by rare disease, including patients and family members, who shared their stories of living with and seeking treatment for their conditions. Guest speakers included Melissa Goetz, co-president of the Familial Chylomicronemia Syndrome (FCS) Foundation, whose daughter has FCS, Jay Honsinger, whose son has been diagnosed with Niemann-Pick Type C disease and Jennifer Rittner, who herself has stiff-person syndrome.
“We are thrilled that we were able to move this legislation along, and we will continue to work until we satisfy the needs of treatment for those that are living with rare and ultra-rare diseases,” said Congressman Tonko. “The HEART Act works to amplify the voices of patients and families living with rare diseases. That amplification is important — to make certain that there is connection, coordination and involvement of families and patients in the FDA treatment review process. As this involvement increases, so does hope — to evaluate treatments that could make a huge difference in the lives of those affected.”
“RNA Institute researchers are working to advance our understanding of and treatments for several rare and ultra-rare diseases including myotonic dystrophy, several forms of spinocerebellar ataxias and inosine triphosphate deficiency – all of which lack FDA-approved treatments,” said RNA Director Andrew Berglund. “We are also strong believers in the power of partnering with patients, their families, advocacy groups and foundations to move these efforts forward. To this end, we recently established a new advisory group called ‘Friends of the RNA Institute’ that brings together individuals affected by rare diseases to ensure they have a strong voice in our mission.
“Working directly with patients and learning from their experiences is critical to the process of developing treatments. The HEART Act will play a vital role in facilitating this sort of exchange as it applies to the full spectrum of rare and ultra-rare diseases.”
[Watch Spectrum's story from Tuesday's event to learn more.]