Leading the Fight to treatand cure Tay-Sachs, Canavan and related diseases
The TSGT Consortium announced in 2011 that it had cured Tay-Sachs in mice, reached a point where affected cats are thriving after treatment, and demonstrated a rare breed of sheep reaping the benefits of
Gene Therapy for treating Tay-Sachs. Treatment proof of concept was demonstrated in May 2011 and feedback from the Food and Drug Administration (FDA), which ultimately approves human clinical trials, has been favorable. The National Tays-Sachs and Allied Diseases Association’s (NTSAD) funding network and the National Institutes of Health (NIH), has supported this work to date with grants of over $5 million. Pledges have also been made for 75% of the additional $1.4 million necessary to reach human clinical trials in 2012.
“2012 is a critical year for us and a time when clinical trials with children for the treatment of Tay-Sachs disease are expected to begin,” said Kevin Romer, president of The Mathew Forbes Romer Foundation, Mathew’s father and the newly elected president of NTSAD. “The progress we are making is fantastic but it’s the generosity of donors and friends of the Foundation that will make the difference and help us find a cure that much faster.”
Dr. Florian Eichler, principal investigator for the Tay-Sachs gene therapy – and one of the individuals working on finding a cure for a number of children’s fatal genetic diseases – joins Kevin and Lisajane Romer, founders of the MFR Foundation; Sue Kahn, Executive Director of the National Tay-Sachs & Allied Diseases Association; as well as the Ogden Family, which includes son Kaleb, who suffers from Sandhoff’s Disease (a rare form of Tay-Sachs). The Ogden family raised $5,500 through their organization, Kaleb’s Cure, and donated it to the NTSAD at “An Evening of Sweetness & Laughter."
An impactful video, now available on The Mathew Forbes Romer Foundation’s YouTube site, led the audience through a journey, which introduced some of the children and families afflicted with Tay-Sachs and other diseases, provided an update on the MFRF mission and its progress, and culminated with an introduction of the gala’s prestigious “See the Light” Awards Ceremony honorees. The Foundation’s two award winners have both made outstanding contributions in pioneering and delivering genetic testing and education programs and moving research forward to eventually find a cure.
Finally, to end the evening with sweetness and laughter, guests were treated to a comedic performance by Cory Kahaney, a regular on The Late Show with David Letterman and Comedy Central, as well as an Off-Broadway star, and a delectable dessert bar.
The wonderful support for this drive to clinical trials was made possible by the generosity of the major sponsors including: Adolph and Rose Levis Foundation, Paul and Judy Levis Markhoff, Lisajane and Kevin Romer, Eric and Karen Schiffer, Baron Delivery on Demand, Grossman and Roth P.A., Tyco Security Products, Carole Romer, Lois and Herm Siegel, Linda and Harry Meran, Temple Beth El of Boca Raton, Pablo and Bettina Meles, Mike and Joan Siegel, Neil Eisenband, and Evan and Joan Deoul.
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 13 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.
Since being launched, the Foundation has:
The MFRF is the South Florida affiliate of National Tay-Sachs and Allied Diseases Association (NTSAD) and a member of the Jewish Genetic Disease Consortium. In addition to pioneering genetic education and screening fairs in South Florida in 2003, MFRF co-founded the NTSAD National Research Initiative in 2002. For more information, visit www.mfrfoundation.org.
National Tay-Sachs & Allied Diseases Association 2001 Beacon Street Suite 204 Boston, MA 02135
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At NTSAD, we are making a difference. Every day. Helping families and individuals find their way and providing hope by supporting research on many levels. All of this takes compassion and determination.
There is no time to waste, and families need our help. Those who are fighting today and those who will seek our help tomorrow.
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