Individually we are one drop.
Together, we are an ocean.
- Ryunosuke Satoro

Friday, April 24, 2015
NTSAD's Annual Family Conference
 
Our 37th Annual Family Conference held in Reston, Virginia last week was a huge success and our largest one to date! There were 78 families attending including 10 new families, 15 Late Onset adults, and many parents, grandparents and extended family and friends. 

Preceding the conference was NTSAD's Science Symposium & Workshop  well-attended by researchers and health professionals. They came away from this meeting energized and ready to invite families to be part of the process next year. A synopsis is below with more to come next week!

At the same time, NTSAD hosted its first New Family Boot Camp to ease new families into the conference with an introduction to terms, issues and each other before the official start of the weekend. It is safe to say that the New Family Boot Camp will be a permanent part of the conference program for years to come.

The rest of the conference program offered families knowledge, support and compassion throughout the weekend. Below are several highlights from the long weekend. If you weren't able to attend, but want to know more please contact Diana Pangonis, Director of Family Services, at diana@ntsad.org or Becky Benson, Conference Coordinator at becky@ntsad.org

We also ask those of you who did attend the conference to take a moment and share your thoughts in this survey here; it will help us when the planning begins for the 2016 Annual Family Conference!
RESEARCH * RESEARCH * RESEARCH * RESEARCH
Below is a brief summary of the NTSAD Science Symposium & Workshop and the NTSAD Annual Conference Research Roundtable Discussions. Stay tuned for next week's  Research Review for more details...
SciSym2015 NTSAD Science Symposium  & Workshop for Researchers and Healthcare Professionals 




This day of discussion gave researchers and clinicians an opportunity to consider ways in which we can converge to make progress together. The program identified critical issues in our current path and fostered collaboration between clinicians, scientists, as well as patients and their families. Moving forward the goal will be to include families to help collect information, which will then be used to help measure and improve patient outcomes and quality of life measurements for future clinical trials. 

In next week's monthly NTSAD Research Review you will learn more about the following topics discussed during the Symposium:

Common Elements of the Pathogenesis of Neurodegenerative Diseases
Clinical Trial Design and Outcome Measures: FDA Perspective
Specific Issues Related to the Gangliosidoses
Specific Issues Related to the Leukodystrophies
Small Molecule Approaches that could be Disease-Modifying
Current Status and Future of Gene Therapy
Newborn Screening and its Role in Developing Therapies
NTSAD's Diseases: Outstanding Issues in Clinical Trial Readiness

R esearch Update Session Roundtable Discussions
The Research Update Session was held in the same format as previous years in rotating roundtable discussions giving families the opportunity to hear about an expanse of research topics.  Next week's Research Review will share more about the topics covered at the Research Update on Friday, April 17th:

Advances in expanded and pan-ethnic carrier screening
NTSAD-funded Lectin-assisted intranasal enzyme replacement therapy 
    for GM1 gangliosidosis
Updates from the Tay Sachs Gene Therapy Consortium for gene therapy      for GM2 gangliosidosis and GM1 gangliosidosis (also NTSAD funded)
Importance of natural history studies, registries, and clinical endpoints for    designing clinical trials
37th Annual Family Conference Award Recipients
Marquardt, William GM1 with family
Ethel Berkman Power of One Award
NTSAD parent, Oralea Marquardt
Nominated by Rose Urban and Shelly Ogden
"Williams' courageous nine year battle with GM1 motivated the start of a pediatric palliative care program in their area.  Williams' life, and the way in which Oralea cared for him continue to be an inspiration to all of our NTSAD families!"

Above and Beyond Award 
NTSAD supporter, Michael Sussman
Nominated by Marion Yanovsky
"Michael has been a constant supporter of the NTSAD's New York Area Chapter over many decades and in addition has also supported NTSAD National. Michael always kept in touch and talked about doing something special for NTSAD to honor the work begun by his parents that
we carry on today."
Candle
NTSAD Commemoration
Families gathered together on Friday, April 17th during the conference to honor loved ones in a moving candle-lighting ceremony. Poems were read and NTSAD sibling Samie DuVall sang a song she wrote in honor of her brother, Nickolas and all the children. The commemoration ended with the planting of pinwheel flowers named for attendees' loved ones. At the same time, a worldwide commemoration slideshow was launched which can be seen here. Our thanks to the NTSAD Commemoration Committee for their thoughtful planning and hard work. 
Our Thanks to All Who Made it Possible!

Amicus Therapeutics

 

Benton's Family Assistance Fund

In loving memory of Benton Auerbach

 

Cameron and Hayden Lord Foundation, 

Affiliate of NTSAD

In loving memory of Cameron and Hayden

 

Canadian Association for Tay-Sachs 

& Allied Diseases (CATSAD)

Wiz Fund

In loving memory of Elizabeth 'Wiz' Gagne

 

Nicole Casty and Jonathan Vignati

In loving memory of their daughter, Mia Alexandra

 

Christine Chapman and William Ohle    

In loving honor of her granddaughter, Emma Artinian

 

Bonnie and Barry Davis

In loving memory of their son, Adam

 

Delaware Valley Chapter of NTSAD

In loving honor of all the children

 

Kathryn and Anson Flake

In loving memory of their nephew, Kyle Klock

 

Genzyme, a Sanofi company

 

Monica and Gary Gettleman

In loving memory of their daughter, Brooke

 

Grossman Foundation

 

Sharon and Steve Hoffman

Harry Hoo Fund

In loving memory of their son, Harry

 

Manita and Debasish Hota

In loving memory of their daughter, Ria

 

Iberdrola Renewables, LLC

In support of Noah Leos and others that 

suffer from Tay-Sachs

 

JScreen

 

Lysogene

 

Staci Kallish and Chris Beer

In loving honor of Meryl Kallish and 

her contributions  to NTSAD on her 65th birthday

Love, Staci and Chris, Matt, Tania, Shaun and Michael

Judy Kaplan, Friends & Family

In loving memory of Linda Kaplan

 

Richard Karl and Bruce Steiger

In loving honor of their daughter, Krystie

 

Sarah Mattingly

In honor of all the families

 

Mary and Paul McDonald

In loving honor of their granddaughter, Cora

 

Mark Mushin

In loving honor of his daughter, Alyssa

Happy 21st Birthday, Alyssa!

 

Sheila and Allen Mushin

In loving honor of their granddaughter, Alyssa

Happy 21st Birthday Alyssa!

 

New York Area Chapter of NTSAD

 

Shelly and David Ogden

Kaleb's Cure

In loving memory of Kaleb Ogden

 

Porter Family and the Jones Family

The Emily Lynn Jones Memorial Fund of NTSAD

In loving memory of their granddaughter 

and daughter Emily

 

Ratner Family

In loving memory of Ilana Ratner

 

Redbook Magazine

In honor of Emily Black and in loving memory 

of her little seal Ronan

 

Mathew Forbes Romer Foundation, Affiliate of NTSAD

In loving memory of Mathew Forbes Romer

 

Laura and Simeon Schindelman

In loving honor of all the families

 

Tikun Olam Program of United Synagogue Youth

 

Cynthia Tifft, MD, PhD and Forrest Hall, PhD

In loving honor of all the families

 

Carol and Eric Zimmerman

In loving honor of Emma Zimmerman

 

We give special recognition to the donors above that have contributed $250 or more.  We are truly grateful to all the supporters of the Annual Family Conference

 

 

Supporting NTSAD Makes a Difference!

We welcome gifts to honor lives gone too soon to support our many efforts to fund research, care for families and their loved ones. Gifts can be made through our website hereThank you for supporting NTSAD and giving hope to all our families!
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Contact Us
 
Sue Kahn, Executive Director ( skahn@ntsad.org )
Joan Lawrence, Development Director (joan@ntsad.org)
Ingrid Miller, Office Manager (ingrid@ntsad.org)
Diana Pangonis, Family Services Director (diana@ntsad.org)
Becky Benson, Conference Coordinator (becky@ntsad.org)
 
 
National Tay-Sachs &  
Allied Diseases Association 
2001 Beacon Street, #204
Boston, MA 02135
(617) 277-4463