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Not
only is it summer, it is the season to start
rallying and raising funds for research for
NTSAD's Research Initiative. Since 2011, over
$132,000 has been raised on and around the
Annual Day of Hope with every
penny going to research.
We're
confident the energy of our supporters will help
us reach our goal of raising at least $50,000 in
honor of our Fifth Annual Day of Hope!
What
is the NTSAD Research Initiative?
The
NTSAD Research Initiative is our comprehensive
strategic research program that funds
cutting-edge efforts to find a cure and promote
scientific collaboration to accelerate these
efforts. NTSAD has supported ground-breaking
research since our inception in 1957 beginning
with identifying the missing enzyme that causes
Tay-Sachs.
In 2002, NTSAD recommitted
itself to finding a cure and founded the
Research Initiative; 47 projects and $3.3
million later in grant awards, we are closer
than ever to a cure. Several projects have led
to larger NIH grants resulting in over $10
Million toward finding a cure!
What
happens when a gift is made to
research?
The
funds designated for research are kept in a
separate
restricted bank account. Every year
a Request for Proposals (RFP) is issued
worldwide, after which proposals are reviewed by
members of our Scientific Advisory Committee and
other research experts. They are evaluated and
scored based on the strength of their plan and
if it fits within the RFP goals. After a final
evaluations, awards are then made.
There
are families who choose to open a Family Fund
that can be designated for a specific purpose.
There are two current family funds,
the Katie & Allie Buryk Research
Fund
and the Maanas Memorial
Fund,
which focus on Late Onset Tay-Sachs and Sandhoff
research, respectively. After consulting with
the Buryk Family, two grants were made to
projects specific to Late Onset research
boosting our research efforts even
further!
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An International Flair to Benefit
Research!
A beautiful dance
performance on June 21st in Ohio by Ria
Mrudula Datla not only moved her
audience, but raised nearly $17,000 for Sandhoff
disease research through the Maanas Memorial Fund named in
memory of her brother who had Sandhoff
disease.
To
see more pictures and to learn more about the
Maanas Memorial Fund, visit our website here. |
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Teeing off
for Research a Success!
NTSAD's
new board president, Brian Manning and his
family held their 12th Annual Drive "Fore" Dylan Golf
Outing on June 19th raising over $30,000 to
benefit the NTSAD Research Initiative. It was a
sold-out event with terrific support from their
family, friends and community as they honored
the memory of their son Dylan who had
Tay-Sachs.
To
see more pictures of the event, visit our
website here. |
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NTSAD
Scientific Advisory
Committee
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Fran Platt, PhD
(Chair)
Jodi Hoffman, MD (Vice chair)
Miriam Blitzer, PhD
Robert Desnick, PhD, MD
Florian Eichler, MD
Mark Haskins, VMD, MS, PhD
Michael Kaback, MD, FACMG
Edwin Kolodny, MD
Heather Lau, MD
Paola Leone, PhD
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Heather
Lau, MD
Paola
Leone, PhDGustavo Maegawa, MD,
PhD
Marvin
Natowicz, MD, PhD
Swati
Sathe, MD, MS
Thomas
Seyfried, PhD
Barbara
Shapiro, MD, PhD
Evan
Snyder, MD, PhD
Cynthia
Tifft, MD, PhD, FACMG
Steven
Walkley, DVM, PhD
Michael
Watson, PhD,
FACMG
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NTSAD Corporate
Advisory Council
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Marion
Howard, MD, PhD (Chair)
Oved
Amitay
Ritu
Baral
Michael
Gladstone |
Kate
Haviland
Joan
Keutzer, PhD
Greg
Licholai,
MD
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Are
you on Facebook and
Twitter?
patient advocacy, and
more. | | |
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For a second year, a Million Dollar
Bike Ride Request for Proposals will be
issued in the coming weeks. A grant for $40,000
will be made to a research project that holds
promise for Sandhoff, GM1, Tay-Sachs, Canavan or
related genetic diseases.
We thank everyone who supported Team NTSAD
on May 9th, 2015, the riders and UPenn's Orphan
Disease Research Center in making this grant
possible! |
The National Organization for Rare
Disorders (NORD), the leading independent,
nonprofit organization committed to the
identification, treatment, and cures of rare
diseases, has announced the availability of new
research grants to study rare
diseases.
The deadline to
submit abstracts and letters of intent is July
31.
They will issue one (1) grant of up
to $30,000 for a clinical research study related
to the diagnosis and/or treatment of Lysosomal
Storage Diseases.
See full RFP and abstract template
here.
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Published
Paper Addresses
Pyrimethamine
In 2009, NTSAD funded an
investigator-sponsored clinical trial at
Hospital for Sick Children and NYU for the
treatment of late-onset with pyrimethamine (PYR)
led by Drs. Joe Clarke and Ed Kolodny. The
results were published in 2011. (An open-label
Phase I/II clinical trial of pyrimethamine for
the treatment of patients).
The study
concluded that leukocyte Hex A activity is
enhanced in vivo by treatment with PYR. However,
future studies were needed to assess the
protocol, including dosage, and perform related
biochemical studies. An Israeli team of
researchers performed similar studies and
recently published their results.
While
they also found that PYR increases HexA activity
in LOTS patients, "the observed increase is
repeatedly transient and not associated with
discernible beneficial neurological or
psychiatric effects."
Visit www.NTSAD.org
to download the paper from the
Library. |
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Today
and
Make a
Difference
Tomorrow |
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