Mathew Forbes Romer Foundation Celebrates New Breakthrough in Gene Therapy Treatment for Tay-Sachs & Other Children’s Genetic Diseases

S. Florida-Based Organization Hosts Annual “Sweetness and Laughter” Gala & “See the Light” Awards Ceremony March 16th in Delray Beach

Boca Raton, FL, January 31, 2019 – Twenty-one years after The Mathew Forbes Romer Foundation (MFRF), a non-profit organization dedicated to the education and research of children’s genetic diseases of the brain, was founded – the organization is finally celebrating the arrival of Gene Therapy as a treatment for Tay-Sachs and other related diseases. After 15 years of research, investments and testing, as a major part of the National Research Initiative co-founded by MFRF and the National Tay-Sachs and Allied Diseases Association (NTSAD), gene therapy is moving into clinical trials and now has the commercial support to drive through to full market availability.

In December 2018, Axovant Sciences, a clinical-stage company focused on innovative gene therapies for neurological and neuromuscular diseases, announced it licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs to address GM1 gangliosidosis and GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) from the University of Massachusetts Medical School.

Terence Flotte, MD, executive deputy chancellor, provost and dean of the School of Medicine at the University of Massachusetts Medical School, and former director of the University of Florida’s Powell Gene Therapy Center, will be honored at MFRF’s Annual “Sweetness and Laughter” Gala & “See the Light” Awards Ceremony. Dr. Flotte is also the principal investigator leading the effort into clinical trials.

Dr. Jeffrey Castelli, Chief Portfolio Officer and Head of Gene Therapy for Amicus Therapeutics, will also be honored at the Gala for the Company’s long-term commitment to people living with rare diseases, as well as its recent acquisition of an extensive gene therapy portfolio of 10 clinical and preclinical AAV Programs in neurologic lysosomal storage disorders, including interests in Tay-Sachs. Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases.

The MFRF will celebrate these honorees and much more at its 21st Anniversary “Sweetness & Laughter” Gala on Saturday, March 16, 2019 starting at 6:00 pm at The Seagate Country Club (3600 Hamlet Drive) in Delray Beach. The CELEBRATION of GENE THERAPY will feature dinner and dessert, an update on Gene Therapy advancements and progress, the “See the Light” Awards presentation, and carry the “Gene” Theme throughout. The attire is JEANS, of course, and the laughter will come from a performance by legendary performer GENE Merola, whose show features audience participation, music and creative improvisation, delighting audiences from South Florida to Las Vegas.

Honorary Chairs Dr. Marcelle and Andrew Rosen will also be recognized for their long-standing support for MFRF. Tickets and sponsorship information are available here. (Early bird pricing through February 16, 2019.)

“This year is extra special for our organization, our donors, and most importantly the families of those impacted,” added Kevin Romer, President of the Mathew Forbes Romer Foundation and past President of the NTSAD. “The families that we impact and the hope they have for treatments and cures is what drives us forward year after year. It is why we started this foundation 21 years ago, in honor of our son Mathew, and it is why we continue to work diligently on our mission.”

MFRF, which is the Florida affiliate of the NTSAD, has raised over $2 million itself to fund projects spanning its mission and vision across three pillars: Research of Treatments and Cures, Education and Genetic Screening, and care through Nursing School Educational programs.

  • Research: Conceived of and grew the National Research Initiative to 61 projects, $3.9 million in grants and many promising therapies with NTSAD. This has led to over $20 million dollars in funding from the National Institutes of Health (NIH) and similar institutions. Helped establish the Tay-Sachs Gene Therapy Consortium to accelerate pre-clinical and clinical trials. Sponsored the Medical and Scientific Sessions at the annual NTSAD Family Conference
  • Screening: Hosted 21 South Florida Genetic Testing and Education Fairs; screened over 1,550 potential parents with as high as 50 percent detection. Produced the training video of the International Tay-Sachs Quality Control Laboratory
  • Nursing: Funded more than 35 nursing scholarships with Florida Atlantic University, Florida International University, and Nova Southeastern University; created nursing school training programs including simulating in-home family care

About The Mathew Forbes Romer Foundation

Inspired by the strength of Mathew Romer, who lost his eight-year battle with Tay-Sachs disease in 2003, The Mathew Forbes Romer Foundation (MFRF) was founded in South Florida 21 years ago with a mission to promote and lead critical awareness, testing, counseling, and research initiatives that hold promise for the prevention and eventual cure of fatal children’s genetic diseases of the brain.

The MFRF is the South Florida affiliate of National Tay-Sachs and Allied Diseases Association (NTSAD), celebrating its own 60-year anniversary, and one of the oldest patient advocacy groups in the country, and a member of the Jewish Genetic Disease Consortium. The president of the MFRF (Kevin Romer) is also past president of NTSAD. The Mathew Forbes Romer Foundation was recognized in 2014 with the Best of Boca Raton Award – Education & Research Foundation. For more information, visit

Melissa Perlman
BlueIvy Communications

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