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Upcoming Symposiums, Patient Day, and Recent GNEM News


"There is immense power when a group of people with similar interests gets together to work toward the same goals."  Wealth for All: Living a Life of Success at the Edge of Your Ability, - Idowu Koyenikan

This year our community has been very busy planning more events globally, as well as in the U.S.A.  We are having Symposiums in Philadelphia, Israel, and a Patient Day in Mumbai, India.  In addition, grants for research have been awarded to researchers to facilitate and accelerate a cure for GNEM.  We are quite fortunate to have our rare disease get the amount of attention that we are receiving.  Please see the upcoming events and news listed below.

NDF Will host Two Symposiums in May, One in Philadelphia, and One in Tel Aviv
The Neuromuscular Disease Foundation (NDF) will host its Sixth Annual Symposium in  Philadelphia.  This symposium has a line-up of  well known GNEM researchers and scientists, including Dr. Huizing from the National Institutes of Health (NIH), D. Klements, Clinical Research Manager, Massachusetts General Hospital, and Dr. M. Lek, NDF Scientific Director.  There will be counseling and physical therapy sessions for patients and family members as well.  Please register here:
https://donate.curehibm.org/event/ndf's-6th-annual-symposium-on-gne-myopathy-in-philadelphia/e226017


The NDF will also host a Symposium in Tel Aviv, Israel on May 29.  Among some of the presenters will be Drs. S. Rosenbaum, Z. Argov, M. Lek, A. Lek, and  M.Huizing. For more information and to register for this Symposium, please see this link:    https://donate.curehibm.org/event/ndf's-6th-annual-symposium-on-gne-myopathy-in-israel-patient-day/e217822

World Without GNE Myopathy (WWGM) Event in Mumbai, India, on June 2, 2019. 
World Without GNE Myopathy (WWGM) will hold a one-day event in Mumbai, India, on June 2, 2019.  For this event GNEM patients, researchers and industry professionals are invited.  Here is a sample of some of the sessions: GNE myopathy patient initiatives and Indian efforts in GNE myopathy, awareness, and treatment; NDF Patient Advocacy and Gene Therapy Progress; and GNE myopathy in the Middle-East.  For more about World Without GNE Myopathy, please visit this site:  http://gne-myopathy.org/#

Recent GNEM News

The NDF awarded 5 grants for GNEM Research and Programs for a total of $700,000.  These grants have been distributed to various researchers, who will  work to speed up GNEM gene therapy. Included in the total grants is also a gift to the National Institutes of Health (NIH) of $150,000 to help with the upcoming ManNAc trial for GNEM patients. For more details, check out this link to access the newsletter.
https://myemail.constantcontact.com/2019-Q1-Report--Hope-springs-eternal--.html?soid=1119021574814&aid=92eqarAlHcg

The NDF Launches a Monthly Patient Huddle

This is a peer to peer virtual support and mentoring group facilitated by GNEM patients. A wide range of topics are addressed at this monthly meet.  In the month of May the focus will be on "Trials and Tribulations of Toileting for Women Living With GNEM."
Please register if you would like to join:  https://curehibm.org/welcome.html

 The World Without GNE Myopathy Launched   A Center for  Drug Discovery (CDD)

 The CDD has been created to speed up discovery of GNEM treatments and other rare genetic diseases.  The CDD plans to fund strategic scientific research where there is a lack of research, in addition to funding targeted research that will bring about a treatment for rare diseases, such as GNEM.

ClinicalTrials.gov
The study details of an Open Label Phase 2 ManNAc in GNEM subjects could be seen at this link:
https://clinicaltrials.gov/ct2/show/results/NCT02346461?cond=gne+myopathy&rank=1

Website Listing Proposed Clinical Sites for Upcoming ManNAc Trial
NN109 Magine
Patients  who are interested in  the upcoming multi-center ManNAc trial may want to monitor this site:  https://neuronext.org/projects/nn109-magine

"We empower patients, build communities & drive forward momentum for rare disease globally."                                                 
I have been attending the Rare Advocacy Summit held by Global Genes for the past five years.  I have learned from researchers, caregivers, and patients on ways to share, empower, and network with my GNEM community.

After attending the first Summit, I came back with renewed hope knowing that I am not alone, my GNEM family is not alone, and that I have "giants" fighting for cures for rare disease patients.  If you are able to make this year's Summit, please register at:
https://globalgenes.org/event/patient-summit/

"The marvelous richness of human experience would lose something of rewarding joy if there were no limitations to overcome.  The hilltop hour would not be half so wonderful if there were no dark valleys to traverse."  Helen Keller





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