2018 has proven to be a year of incomparable achievement for NDF! In addition to coordinating scientific strategies, hosting an impressively attended Symposium at UCLA in August, bolstering programs for our patient community, and being invited to present at prestigious industry related conferences worldwide, we are most proud of NDF's most successful gala to date.
See pictures of 2018 events here.
W
e have worked hard to qualify for, and earn a matching grant of $2.5M
from
a private, non GNEM related foundation who has chosen to work with us
because they believe in us, our strategy and our science
.
Learn more about the grant
here.
While raising the matching funds is a lofty goal, we believe through a combination of multi-year pledges from high net worth donors, grants from a handful of smaller foundations and the generosity of our patients and their friends and families, that we can meet this goal so the opportunity of receiving this grant will not be missed.
We are excited to announce that
we have already raised approximately $1.7M in 2018 and are currently working hard to raise the additional $ 800k needed to reach our goal. Given where we are now, we really do believe that, with help from all of you, we can be even more successful in 2019.
As part of the $2 million in clinical research we have funded to date, we have already begun work in the process of Gene Therapy with
Whole Genomic Sequencing
pre-clinical studies in tandem with our Scientific Advisory Committee, our Scientific Directors, NIH and Perkin Elmer. We have pulled together the best scientists and GNEM experts all over the world to ensure the greatest potential to succeed. In keeping with our strategy for our best chances at getting funding for Gene Therapy all the way to market, we are talking to several biotech companies who have shown interest in collaborating with us.
All of us at NDF thank you for your participation and support this past year and wish all of you a happy and healthy 2019.
Lale' J. Welsh
CEO, NDF
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See photos from the event
here.
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Gala for GNEM with the support of Women Artists is a big win for all!
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Our Gala for the Arts saw over 450 attendees, and many viewers through our live-stream. Through the event, we were able to raise just over $1 million towards our goal of $2.5 million, making for a total of $1.7 million raised so far through grants, sponsorships, donations, and pledges. This means we are only $800k away from our goal! Help us reach our goal.
You can still
donate now
.
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Scientific Research Efforts
In tandem with our impressive consortium of international scientists and industry partners, we continue to support the work of the NIH through gifts earmarked for ManNAc, the closest potential treatment for GNEM patients, with plans for Phase 3 in 2019. Stay tuned for clinical trial updates
here.
Our preclinical studies for Gene Therapy continue with the support of our team at Yale and our Scientific Advisory Committee. Stay tuned for results of our Whole Genomic Studies and other efforts in 2019!
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Challenge Grant Status
NDF has achieved a near-impossible goal in the world of rare diseases: a
$2.5 million challenge grant
from a local, private foundation to support NDF’s goal of developing gene therapy for GNE Myopathy. We are just 800k away from funding gene therapy for the GNE Myopathy community! Learn more and see how you can make an impact
here
.
A big thanks goes out to all who made this possible, including our Philanthropist of the Year, Mr. David Dahl, photographed above with our first patient, Jennifer.
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5th Annual Symposium on GNE Myopathy
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Our Symposium continues to grow.
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Patients, family members, caregivers and scientists from all over the world attended the 5th Annual Symposium on GNE Myopathy at UCLA this year. The Symposium was shared live online and reached more than 17,000 people.
See photos and sessions from the event
here
.
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Our second class of Certified Patient Advocates (CPAs) received pins and certificates at our Symposium
in conjunction with their commitment to help us in our efforts to bring
programs to their fellow patients and caregivers in their home countries.
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Several leading GNE Myopathy advocates, researchers, and industry leaders took the stage at the Symposium for an unprecedented sharing of information, which lead to many progressive dialogues.
Guests speakers, scientists and patients are now represented in 23 Countries!
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The office of L.A Mayor
Eric Garcetti
visited the Symposium to present us with a Certificate of
Congratulations for our efforts in the GNE Myopathy Community!
Thanks to our Certified Patient Advocates and Program Managers, Tara & Amy for their continued outreach efforts!
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International Certified Patient Advocacy Program
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NDF is proud to enlist dedicated GNE Myopathy patients from around the world to assist us with our mission of patient advocacy worldwide. Our Certified Patient Advocates (CPAs)
speak many languages and are therefore able to reach out to more patients (and potential patients) in their home countries on behalf on NDF to introduce them to our community and resources. These CPAs are committed to help NDF in our efforts to bring programs to patients and families in their respective countries. Their mission is aligned with the NDF’s goal to enhance the quality of lives of GNEM patients and their families. L
earn more about our CPAs
here
.
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With the support of the NDF, our CPAs host Patient Days in their respective countries. Pictured above is a Patient Day held in the
UK, also pictured are Patient Days held in Turkey and Italy! Because of events like these, hundreds of people across the globe have learned more about GNE Myopathy.
Learn more about past CPA Patient Days
here.
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GNEM Patient Meeting in Italy
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Give Gifts That Give This Holiday
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Shop more GNEM awareness gear
here.
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This holiday season give gifts that give! Purchase GNE Myopathy awareness gear and proceeds will go towards funding NDF's mission driven programs! Shop
here
.
Give A Donation In Someone's Honor
We may have raised $1.7 Million, but we still have $800k to go before we meet the
$2.5 million dollar challenge grant! Help NDF reach our goal and give a special gift this holiday by donating in someone's honor
here.
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Painless Giving At Little To No Cost To You
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Support NDF's Mission Monthly
Support the GNEM community by giving through NDF on a recurring basis. A monthly donation starting as
low as $26
can be set up through our monthly giving program. Click
here
to help
empower NDF
overcome obstacles and continue to move ahead assertively with our mission.
Donate stock, cars or boats.
We've set up an account to receive your gifts of stock
in one easy transaction. For more details, please contact us
here.
Donate Your Birthday
Set up a fundraiser through Facebook for NDF during your birthday month. Ask your friends to donate to a cause you love as a gift to you! Click
here to learn how.
Shop AmazonSmile
Support the Neuromuscular Disease Foundation this holiday season by using
smile.amazon.com.
The AmazonSmile Foundation will automatically donate 0.5% of the purchase price at no cost to you each time you make eligible purchases. Just type in "Neuromuscular Disease Foundation" as your charity of choice and Amazon will do the rest.
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Support. Volunteer. Advise.
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We can't do this alone. Are you be interested in being part of the solution to GNEM by giving some time and energy to help further our efforts?
Please consider volunteering for NDF or serving on a committee or our Board. NDF is looking for a wide range of volunteer help, from gala leadership to new committee chairs, and possible (paid) contract positions. To get involved, email our CEO,
here
.
New Board Elections will take place and be announced in Q1, 2019.
Don’t have time to volunteer, but still want to help?
Click below to donate now.
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NDF's mission is to enhance the quality of the lives of people living with GNE Myopathy through advocacy, education, outreach and funding critical research focused on treatments and a cure.
If you like what we do for your community, please consider getting involved in a way that's meaningful for you
here.
We are social!
Click below to follow us.
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Los Angeles
269 S. Beverly Drive, Suite 1206
Beverly Hills, CA 90212
(310) 721-1605
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New York
10 Bond Street, Suite 330
Great Neck, NY 11021
(310) 721-1605
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