Leading the Fight to treatand cure Tay-Sachs, Canavan and related diseases
THE MATHEW FORBES ROMER FOUNDATION, NTSAD's South Florida Affiliate, CELEBRATED ITS 15 YEAR ANNIVERSARY WITH DINNER & SHOW, AWARDS CEREMONY AND KICK-OFF OF NEW NURSING PILOT PROGRAMFoundation is Focused on Education & Research of Children’s Genetic Diseases of the Brain; Event Celebrated Honorees from Florida Atlantic University, Florida International University and the University of Florida
Boca Raton, FL, March 3, 2014: The Mathew Forbes Romer Foundation (MFRF) celebrated its 15th anniversary with “An Evening of Sweetness & Laughter” yesterday, Sunday, March 2, 2014 at the Broward Center for the Performing Arts in Fort Lauderdale, Florida. The event included a matinee show featuring American political satire group The Capitol Steps; a “See the Light” Awards Ceremony honoring three leaders of the fight against children’s genetic diseases of the brain; as well as dinner and dessert.
“An Evening of Sweetness & Laughter” and its prestigious “See the Light” Awards Ceremony recognized three individuals from academic institutions throughout Florida, who have led critical initiatives dedicated to the caring and curing of children’s genetic diseases of the brain. The honorees were:
• Dr. Thomas J. Conlon: Director, Powell
Gene Therapy Center Toxicology Core, University of Florida College of Medicine;• Dr. Marlaine Smith: Dean, Christine E. Lynn College of Nursing, Florida Atlantic University; and• Dr. Ora Strickland: Dean, Wertheim College of Nursing and Health Sciences, Florida International University.
Dr. Conlon is renowned for ensuring the safety of Gene therapy as the final approval before clinical trials. Due to the diligence of his toxicology team, a major safety risk was averted which in turn increases the probability of successful upcoming clinical trials with affected children. Additionally, Drs. Smith and Strickland currently head innovative nursing school programs at their respective universities and have partnered with The Mathew Forbes Romer Foundation for the education and care portion of its mission.
“This year’s honorees each individually speaks to the importance of both research and education as we as we converge on finding a cure for Tay-Sachs and other children’s genetic diseases,” said Kevin Romer, president of The Mathew Forbes Romer Foundation.
The event also served as an opportunity to introduce a new Nursing Pilot program developed with The Mathew Forbes Romer Foundation, Florida Atlantic University and Florida International University. As part of the pilot program in Palm Beach County and Dade County, nursing students are being placed in homes, where they are assisting families with affected children. The nursing students will help the families with care plans and support as part of their clinical training. This pilot program is a natural progression from the many nurses who have received scholarship money from the Foundation in past years. The Mathew Forbes Romer Foundation strongly believes in the importance of preparing nurses for in-home care, where many children with fatal genetic diseases need to be cared for continuously.
“There continues to be a shortage of nurses within in-home care,” added Romer. “For those families dealing with the medical and emotional strains that caring for a child with a genetic disease brings, having a nurse with the right training and compassion in the home just a few hours a week can be life changing.”
“We are very excited about the potential of this program and ultimately expanding it to more families in South Florida,” added Dr. Smith. “From an academic standpoint, this program is invaluable to our students as they have the opportunity to learn so much from the families and parents who have been acting as primary caregiver for years. From a health care perspective, home care is the future of nursing and the at-home and community-setting care experience is more critical than ever.”
About The Mathew Forbes Romer FoundationInspired 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 15 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: • Co-Founded the National Research Initiative with the National Tay-Sachs and Allied Diseases Association (NTSAD);• Helped raise almost $3M of funding, which has supported over 40 research projects related to Tay-Sachs Disease and been complemented by over $7M of federal funding to use towards work with the National Institutes of Health;• Hosted countless genetic testing and education fairs around South Florida and reached more than 1,000 people through screening efforts;• Expanded screening panel to include 18 genetic disease tests (three times as many than when the MFR Foundation was founded);• Created and grew over 25 pediatric nursing scholarships across five colleges and universities; and• Partnered with numerous organizations in South Florida and nationally to drive its mission.
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, MFRF co-founded the NTSAD National Research Initiative in 2002. For more information, visit www.mfrfoundation.org.
On January 29th, the Senate passed the Newborn Screening Saves Lives Reauthorization Act (S.1417) by unanimous consent! NTSAD signed onto the letter and supports this legislation with the hopes that one day when treatments are available for our group of diseases, children born with rare genetic diseases will have a brighter and healthier future.
On March 30, 2013, H.R. 1281, the Newborn Screening Saves Lives Reauthorization Act was introduced in the U.S. House of Representatives and referred to the House Committee on Energy and Commerce. A companion bill, S. 1417, was introduced in the U.S. Senate on August 1, 2013 and referred to the Health, Education, Labor and Pensions Committee.
The Newborn Screening Saves Lives Reauthorization Act reauthorizes critical federal activities that assist states in improving and expanding their newborn screening programs, supporting parent and provider newborn screening education, and ensuring laboratory quality and surveillance.
Summary of key provisions
Visit Save Babies Through Screening website to find out about your state's screening and to learn more.
JScreen, a new program launched in early September 2013 by Emory University in partnership with the Marcus Foundation, turned to NTSAD this past year for our guidance as they developed their program to expand carrier screening for the Jewish community and to give families of Jewish descent “easy access to information and allow testing to be done conveniently from home.” They wanted to be sure they offered correct and current information about Tay-Sachs screening before launching to the public. NTSAD’s Scientific Advisory Committee (SAC) made suggestions regarding testing and JScreen made it their priority to heed their advice about education and access to
enzyme testing for Tay-Sachs carrier screening. (On the recommendation of our Scientific Advisory Committee, JScreen will track the percentage of participants that are following up with enzyme testing and will keep NTSAD apprised of the results.)
Karen Grinzaid, JScreen ‘s Senior Director of Outreach Initiatives, attended the Annual Family Conference and Scientific Advisory Committee (SAC) meeting to present JScreen’s plans for ensuring that their Tay-Sachs screening would be performed according to NTSAD’s Tay-Sachs Testing Guidelines.
Their Tay-Sachs enzyme plan for program participants includes:
JScreen, is currently up and running in Georgia, Florida, North Carolina, South Carolina, Washington DC, Virginia and Maryland, with hopes for national expansion in the near future. Visit their website at www.jscreen.org for more information.
This unique project evolved from years of successful youth retreats at the annual family conferences, and engaging healthy siblings whose brothers/sisters have/had Tay-Sachs, Canavan, GM1, Sandhoff and related genetic diseases. After seeing his mom involved in the "Parenting a Child with Life-limiting Illness," Sebastian, whose sister had Tay-Sachs, felt strongly that siblings should be heard through this medium as well. The plan is to create a web based video series designed to support siblings of children affected with life limiting illness, as well as those living with complex and chronic conditions. Specifically, the project will look to support children between the ages of 6 and 18 years of age. The hope is that this series will:
Videos will ultimately be promoted using social media channels such as You Tube and Facebook and will be accessible by the broader rare disease community.
NTSAD received another Genzyme Pal Award in 2011 that allowed NTSAD to produce the "Parenting a Child with Life-limiting llness" DVD film and accompanying guide which became available in April 2013. To read more about that project, click here.
The Genzyme Patient Advocacy Leadership (PAL) Awards is a global grant program that supports outreach programs by non-profit organizations that work on behalf of patients living with lysosomal storage disorders (LSDs), a group of rare, inherited disorders that cause progressive and debilitating health problems.
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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.
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