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Take Action Now!
Ask your representative to co-sponsor
the
OPEN ACT HR 971 to help bring
treatments to rare disease patients!
The
EveryLife Foundation
just launched an ACTION ALERT to enable individuals to write their representatives and request that they co-sponsor the bill. To date, over 100 messages have been sent to Congress and the bill has seven (7) co-sponsors to date.
Cosponsors to date
Rep. McCaul, Michael T. [R-TX-10]*
Rep. Butterfield, G. K. [D-NC-1]*
Rep. Poliquin, Bruce [R-ME-2]
Rep. Graves, Sam [R-MO-6]
Rep. Grothman, Glenn [R-WI-6]
Rep. Fortenberry, Jeff [R-NE-1]
Rep. McMorris Rodgers, Cathy [R-WA-5]
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RESEARCH @ NTSAD's Annual Family Conference
A Research Sneak Peek
The Annual Family Conference will be held in exactly three weeks and the anticipation for all it offers is palpable.
Prior to the official start of the Annual Family Conference, on Thursday, April 16th,
NTSAD will be hosting a Science Symposium & Workshop for researchers and health professionals. Cynthia Tifft, MD, PhD of NIH and Fran Platt, PhD of Oxford University are the co-chairs of this impressive symposium. The goal is to bring together researchers and healthcare professionals to discuss a number of topics related to the lysosomal storage and leukodystrophy diseases, and incorporate workshops that address NTSAD's Clinical Trial Readiness Research Strategy. We are pleased to have highly-regarded speakers including Yao-Yao Zhu, Md, PhD, from the Center for Biologic Evaluation and Research, FDA. An agenda for the meeting can be found
here.
The
Annual Family Conference Research Session, held Friday, April 17th, will offer families and individuals affected by the lysosomal storage and leukodystrophy diseases the opportunity to learn more about the work funded by NTSAD. Speakers include
Mimi Blitzer, PhD, David Radin, PhD, Doug Martin, PhD, and Gerald Cox, MD. They will discuss updates in carrier screening, the importance of natural history studies to research, and gene therapy studies for Tay-Sachs, GM-1 and Canavan diseases. Families will have the opportunity to hear David Radin, PhD discuss BioStrategies NTSAD-funded research on "Lectin-assisted transnasal delivery of corrective enzyme for GM1 gangliosidosis." Read more
here.
Other
experts available to speak with at the conference include Dominic Gessler, MD, Jeanine Utz, PharmD, Florian Eichler, MD, and more.
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FAMILY SUPPORT
Gearing Up for the NTSAD Family Conference 2015
The
Annual Family Conference
is three weeks away where over 75 families will come together for a long weekend of support, information, and hope. A year of planning with over a dozen volunteers and close to 350 donors has made the conference possible. Nearly $50,000 in Helping Hand Grants has been awarded to 56% of the families to help offset some of the expenses in attending this year's conference.
For anyone that has given a gift in support of the Helping Hand Grant Fund, know that the conference sessions give families and individuals the tools to care for their loved ones, themselves and the opportunity to connect with others. It is the one time of year where they can feel "normal;" and that includes the healthy siblings, many of whom have grown up together.
See what your generosity means to families
here.
Be a Part of the Conference!
A gift is a wonderful way to support the
conference experience, the families, their children and the adults affected by these rare genetic diseases.
It's not too late to sponsor the conference! Opportunities range from underwriting a
particular session to either sponsoring an entire meal or simply a table. All sponsors are recognized in conference materials, NTSAD's website and at meals.
To learn more, visit the NTSAD Sponsor Hope page
here, or, contact Joan Lawrence, Director of Development, at
[email protected].
[The conference] is the one place I look forward to going every year
where I can be with my family and it's OK to laugh and cry at the same time -
where I
don't have to say anything,
everyone knows and
understands exactly how I'm feeling.
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QUILTS OF HOPE
Buy a Chance to Win a Quilt of Hope
Every $5 ticket bought is a chance to be a part of this great tapestry known as the NTSAD
family.
These quilts were handmade by families who have been touched by a rare genetic disease so it is quite safe to say that the love for their sons, daughters, grandchildren, nieces and nephews are woven into these quilts.
Every dollar raised through this Quilts of Hope raffle will benefit the Helping Hand Grant Fund that gives families some financial assistance in attending the annual family conferences.
Support hope and buy a ticket, or two, or three....
here
.
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MOST CREATIVE FUNDRAISING...UPDATE
A big thank you to Xavier's dad, Lucanor, for organizing our most creative fundraiser to date! Over the course of several weeks, he raised nearly $4,000 for NTSAD while he shared the different stages of his haircut.
We salute Lucanor, his family and most of all, Xavier, for raising awareness and funds that will help immensely as we continue the work to care for families and find a cure.
If you would like to make an additional gift to honor
Xavier, click
here
.
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Mark your calendars for these future NTSAD events spanning the East Coast! Stay tuned for more details as they become available.
Licht-Sussman Friend-raising Event 
Washington, D.C.
June 11, 2015
Drive for Dylan Golf Outing
Honey Brook, Pennsylvania
June 19, 2015
Fifth Annual Day of Hope
Worldwide!
September 19, 2015
Imagine & Believe
Boston, Massachusetts
November 5, 2015
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