“The NTSAD Family Conference has always been about the families of those affected with life-threatening genetic diseases,” said Sue Kahn, Executive Director of the NTSAD. “The four-day event is designed to give families updates on research findings, practical advice on how to care for infants to adults affected by these diseases, support from mental health professionals as well as those families who have been in a similar situation, and most importantly – hope.”
The conference features a robust schedule with activities for affected children, their parents, grandparents, and their siblings, as well as for affected adults. Family photography sessions will also be made available throughout the four-day event.
• Beginning on Thursday, April 19th, families are invited to get to know one another and attend the welcome dinner in the evening.
• The Friday, April 20th schedule features support group discussions, updates on the latest research breakthroughs in gene therapy (shared in conversational terms), as well as a commemoration ceremony for those children/family members who have passed away.
• The Saturday, April 21st program features numerous sessions including “Symptom Management for Affected Children,” “Speech Therapy and Choking Prevention,”
• Finally, Sunday, April 22nd will mark the conclusion of the conference and offer attendees a brunch send-off as well as the opportunity to join with other families in attending the local Orlando theme parks.
Orlando, Florida based family, the Ogdens, will be among the nearly 100 families attending the conference. Returning to the conference for a second time, parents Shelly and Dave will be joined by Grandma Penny Shesa and three-year-old Kaleb, who has Sandhoff’s Disease, a rare illness similar to Tay-Sachs. “We met some amazing people at the conference two years ago when we attended only three-weeks after Kaleb’s diagnosis – people who have walked in our shoes, who had lost their child to one of these diseases and people who are walking in our shoes now,” said Shelly Ogden, Kaleb’s mom. “It's nice to be in a room full of people for once and feel normal and understood. We are also looking forward to the research updates but know at this point it’s too late for our Kaleb.”
The conference’s research update, which will take place Friday afternoon, is sponsored by the Florida NTSAD affiliate, The Mathew Forbes Romer Foundation (MFRF). Kevin Romer, president of the MFRF, is also the current NTSAD board president. “Since beginning our NTSAD research initiative 10 years ago, we have been looking forward to the day when we would be able to treat children with these therapies,” said Romer, who is also Mathew’s father. “We expect to be in clinical trials in later this year thanks to the continued support of our donors as well as the community and researchers.”
The Tay-Sachs Gene Therapy (TSGT) Consortium supported by the NTSAD Research Initiative, 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 NTSAD’s 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 the additional $1.25 million necessary to reach human clinical trials in 2012 - with the NTSAD’s next focus on the funding necessary to complete Phase 1 and 2 Trials.
To learn more about the 34th Annual NTSAD Family Conference or to attend, please visit http://www.ntsad.org/
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National Tay-Sachs & Allied Diseases Association (NTSAD) is one of the oldest patient advocacy groups in the country. The organization focuses on funding research, supporting over 500 families and individuals worldwide, and raising awareness to prevent disease. Today, NTSAD is recognized as a leading non-profit patient group with a demonstrated commitment to service, science and support. NTSAD gives help and hope to thousands of individuals and families from many backgrounds and ethnicities affected by Tay-Sachs, Canavan and related genetic diseases all over the world. The programs and services offered by the NTSAD include: Family Services, Research, Education and Advocacy. Learn more at www.NTSAD.org.