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Summer Edition #2
NDF NEWSLETTER
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July 2017
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Our Website
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Quick Links
UPCOMING NDF EVENTS:
Please check our site for updates.
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Please support our sponsors:
Ultragenyx
Beverly Hills Rotary
Global Genes
IAJF New York
News Flash:
The Iranian American Jewish Federation (NY) now directly supports NDF's efforts towards gene therapy!
Want to be part of our solution? Check out the many ways in which you can join our global effort for a cure by clicking here.
Symposium 2016 Photos
Patients, Family, and Friends
Speakers
For more event pictures visit Curehibm.org
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NDF's Impact
"NDF has not only raised awareness about HIBM, but has played a central role in bringing researchers of diverse background from all over the world together to collectively tackle this devastating disease. NDF has also promoted greater communication between researchers, patients and patient advocates, bringing a sense of community to those who suffer from the disease and the people who care about finding a cure."
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Siavash Kurdistani, MD, Professor and Chair, UCLA Department of Biological Chemistry
"I am thankful for NDF's help in eliminating the isolation felt by patients and for providing vital information to us and our caregivers. Their website and events are relevant and engaging. Most importantly, NDF empowers patients and carers to keep driving for change and to never give up hope."
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Mona Patel, GNEM Patient, Manchester, England
"It is invigorating seeing that NDF tirelessly works towards a cure and is a constant advocate for the GNE Myopathy patients throughout the year, every year. Thank you NDF for never giving up on reaching for a cure and being a champion for GNE Myopathy patients around the world.
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- Debbie Scott, GNEM Patient, Irvine, California
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Please forward this email to a friend who needs to know about NDF!
Gala Beverly Hills
Check out more event photos on our site.
NDF Symposium 2016
NDF Staff, Volunteers & Ambassadors
wish you a healthy, happy summer! |
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Here at NDF, things are moving fast. We want to keep you in the loop and up-to-date. This newsletter highlights some of the exciting news about our collective progress.
In the meanwhile, if you haven't already registered for our upcoming
FREE 2-day symposium on GNE Myopathy at UCLA, you can do so and
learn more here.
I look forward to your comments and feedback as always.
Lale' Welsh
Executive Director, NDF
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NDF Releases 2017 Impact Report
NDF has released its 2017 Annual Report and Income Statement based on 2016 tax filings. Take a look at the entire report to see how your donations are spent, the impact each program has had, and read testimonials from the people we serve.
NDF values transparency. The top pie chart indicates the different types of income realized by NDF. The bottom pie chart indicates NDF's distribution of funds. Over 90 cents of every dollar donated goes straight to funding research and programs providing resources to families living with GNE Myopathy. NDF prides itself in its financial dedication to the GNEM Community.
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Introducing New Members to NDF!
Nancy Lurie, Tara Voogel, Amy Curran , and Jonathan Pizzi have joined the Neuromuscular Disease Foundation (NDF), the world's largest GNEM (HIBM) patient advocacy group. Nancy Lurie is NDF's new Associate Director. Tara and Amy are Patient Advocate Program Managers. Jonathan Pizzi is a Caregiver Liaison with vast experience as a caregiver for patients with GNEM.
To learn more about our entire Board click here:
Who We Are
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NDF Initiative Plans for an International Scientific Consortium
 
With various scientific teams studying GNEM all over the world, NDF has set the ambitious goal for all respected scientists to share their research with one another. Worldwide collaboration will prove more beneficial to patients than would singular studies. NDF's goal is to help bridge gaps in collective knowledge about GNEM. This initiative, spearheaded by NDF, is working toward its goal of coordination between scientists globally with the formation a collaborative consortium for data sharing. Researchers will be able to build upon the latest collective information to avoid dead ends in our quest for treatments and a cure.
Scientists from NIH, Ultragenyx, Hadassah, U.K. and Japan, among many others, will be convening in August at the Symposium hosted by NDF, at UCLA.
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Upcoming Events
August 24 & 25, 2017 - NDF Symposium on GNE Myopathy will be held in Los Angeles, CA
Please join us for our 4th Annual Symposium on GNE Myopathy, a two-day, fact and fun-filled event including up-to-the-minute information, presentations, panel discussions, and individualized breakout groups for patients; caregivers; and scientists, physicians, and industry professionals. Meals will be provided for all attendees.
At the Symposium, patients, family, and friends of GNEM are invited to participate, learn, and communicate with members of NDF, the scientific community, and other industry agencies. A limited number of travel scholarships are available for patients with GNEM to attend the Symposium. See the event invitation here:
NDF Symposium on GNE Myopathy
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For any questions regarding the Symposium, email us here.
Fall 2017 - Standby for our event announcements!
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Dr. Jerry Mendell and NDF's
Gene Therapy Update
We are pleased to announce that Dr. Jerry Mendell from Nationwide Children's Hospital of Ohio, is
NDF's newest partner in gene therapy for GNEM. Through a series of tests on mouse models and building upon NDF's funding of Hadassah Medial Center & Dr. Stella Mitrani-Rosenbaum's research, Dr. Mendell will be moving forward with a meeting at the FDA
(Food and Drug Administration) this summer.
So, what does this mean for patients? A "proof of concept" designation is a step in the right direction for Dr. Mendell's getting approval from the FDA to
begin conducting safety studies on humans.
Dr. Mendell plans to attend our upcoming symposium this summer to meet with patients, scientists and NDF supporters. To learn more about gene therapy & Dr. Mendell,
click here, and you can
register for our symposium here.
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GET SCREENED AT HOME!
GNE Myopathy added to the JScreen Test
JScreen, in association with Emory University, is a non-profit organization that gives individuals and families easy access to information on genetic diseases and provides the opportunity for genetic screening to be done conveniently from home. The screening is completed at home through a saliva sample that the participant collects and ships to the testing laboratory. Genetic testing is completed on DNA collected from the saliva sample. The JScreen panel is an option for those with Jewish and non-Jewish ancestry.
The JScreen panel includes more than 200 genetic diseases, and includes those that are commonly found in the Jewish population. Our Executive Director, Lale' Welsh, worked closely with JScreen and Emory University to get GNE Myopathy listed among the diseases tested for in their panel. To view the full list of diseases that JScreen tests for, click here.
-All information about
JSc
reen
was retrieved from their website.
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NDF member joins MDA's
National Community Advisory Committee
We are proud to announce that NDF Patient Advocate Program Manager Amy Curran has been selected to serve on The Muscular Dystrophy Association's (MDA) National Community Advisory Committee.
MDA's National Community Advisory Committee (NCAC) is a 15-member group that advises MDA on matters of importance to families living with neuromuscular diseases and helps inform MDA's efforts to support individuals to live unlimited lives.
Amy's goals for her work on the committee includes her dedication to increasing awareness for GNE Myopathy and neuromuscular diseases, in general.
To read more about the committee, please visit the
NCAC
website.
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Introducing
Patients' Corner
In each newsletter going forward, we will include a section dedicated to patient and caregiver outreach. Here, you will find links to our website with interesting information just for patients from patients and about patients and their journeys.
GNEM patient and NDF Patient Advocate Program Manager, Tara Voogel, writes a blog,
Tara Talks GNE Myopathy, where she discusses topics relevant to patients, caregivers, and friends affected by GNE Myopathy. Sometimes. a guest will post a blog about their GNEM journey. Amy Curran has recently posted about going to Capitol Hill to advocate for all persons with a disability in a post called ...
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Donate to NDF at no cost to you through your AmazonSmile
If you make purchases through Amazon, you can support NDF by signing up for AmazonSmile. AmazonSmile works in the same way Amazon does with the same offers and prices, while the site donates a percentage of the purchase price to a charitable organization of your choice at no cost to you.
Go to smile.amazon.com and select The Neuromuscular Disease Foundation as your charity of choice.
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Lots of easy ways to help NDF
We've learned something about you in ten years, and that's that you, our supporters, are a mix of all stakeholders in this insidious disease. You are our patients, carriers, family members, caretakers, volunteers, sponsors and donors alike. Each of you has done what you can to demonstrate loyalty year after year. We thank you and let you know that, while we still need your support, we've done what we can to making giving easier.
Please click here
to find out about our annual giving levels and to learn about giving incrementally throughout the course of the year at a level that fits within your family's budget.
Every dollar counts!
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