National Tay-Sachs & Allied Diseases Association Hosts First of Its Kind Drug Development Meeting for GM2

2024-02-01

BOSTON, Feb. 1, 2024 /PRNewswire/

-- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.

EL-PFDD meetings give the U.S. Food and Drug Administration (FDA) and other key stakeholders, including medical product developers, health care providers, regulatory agencies, an important opportunity to hear from patients and their families about the symptoms that matter most to them, the impact the disease has on patients' daily lives and their experiences with currently available treatments. This input can inform FDA's decisions and oversight during drug development and their review of marketing applications.

Nearly 30 people will share their stories and experiences with infantile, juvenile, or late onset GM2, including adult patients, parents, caregivers, and bereaved family members. Other presenters include Jacqueline Karp, MD, U.S. FDA Center for Drug Evaluation and Research; Florian Eichler, MD, Massachusetts General Hospital; Cynthia Tifft, MD, PhD, National Institutes of Health; and Kathleen Flynn, NTSAD CEO.

"NTSAD was founded by families. They are the experts on living with these devastating rare, genetic diseases, and their voices should be heard," said NTSAD CEO Kathleen Flynn.

This virtual meeting will be livestreamed via YouTube, open to the public, free to attend, but advance registration is required. There will be opportunities to call in, submit comments, or participate in live polling. To learn more about the GM2 EL-PFDD meeting, register to attend, or submit comments, visit www.ntsad.org/gm2pfdd.

Meeting sponsors include Sanofi, Cure Tay-Sachs Foundation, New York Area Fund, Mathew Forbes Romer Foundation, Azafaros, and JCR Pharmaceuticals.

About NTSAD

One of the oldest patient advocacy organizations, NTSAD supports families and individuals globally through one-on-one support, mentoring and peer groups, information and resources, and its Annual Family Conference. NTSAD also advocates for patients and advances research. Since 2002, the organization has awarded more than $4.7 million in grants that attracted more than $30 million in research investments, leading to the development of potential therapies for Tay-Sachs, Canavan, GM1, and Sandhoff diseases. For more information, visit NTSAD.org.

SOURCE National Tay Sachs and Allied Diseases Association Inc

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