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NDF Symposium on August 24 & 25, New Brochure for World Without GNE Myopathy, Newly Formed Group of Turkish Patients, The NIH Site to Monitor for Phase 3 ManNAc Trial

                                                    NDF 2016 Symposium photo
                                         
The Neuromuscular Disease Foundation

The Neuromuscular Disease Foundation (NDF) is gearing up for a full two days' program for the fourth annual symposium. All the major stakeholders involved in GNE Myopathy, including patients will be attending this impressive and highly informative Symposium.  Here are some of the highlights and a link to the agenda:  
  1. The largest symposium to date on GNE Myopathy (GNEM)
  2. Attendees,patients, and scientists from 15 different countries, 
  3. Scientists and researchers who will present at this symposium will include include all the major players in GNEM,  such as those from National Institutes of Health, Ultragenyx, Perkin Elmer, Nationwide Children's Hospital in tandem with Ohio State University, UCLA, Hadassah Medical Center- Israel, Japan, Korea, UK, just to name a few, and a variety of leading physicians in the field of neurology.
  4. The NDF has formed a "Scientific Consortium on GNE Myopathy" led by its Executive Director and Board to encourage and expedite sharing of data on GNEM so that we could move towards a cure.  
  5. The NDF is maximizing the potential of this gathering in order to streamline issues that affect not only scientists but also patients, their caregivers and family members, which go often unsupported. The NDF is accomplishing these important goals through special programs by addressing health, nutrition, psychological aspects of the patients and advocacy.
  6. The NDF is recruiting and training 8 "Certified Patient Advocates" to help create awareness and action by cohosting GNEM Patient Days in their hometowns and respective countries. 
  7. The NDF is very sensitive and attentive about the needs of rare disease patients. They have listened to patients suggestions and is offering sessions for "patient-to-patient" only, and a separate session  for family/caregivers.
  8. The NDF is generously paying for travel scholarships to many patients, as well as providing lodging and meals for patients coming from many foreign countries and within the U.S. 
  9. NDF is supported for this Symposium  in part by Ultragenyx, public donations including patients, doctors, and board members.  It takes a village, including you, to fund  a cure for GNEM.     Please consider "Joining the Global Effort to Cure GNE Myopathy and make a donation to:  http://curehibm.org/get-involved
  10. Link to current agenda for the Symposium
  11. Link for NDF Facebook page to follow live streaming of the Symposium.
World Without GNE Myopathy, India
World without GNEM (WWGM) is diligently working to generate awareness and understanding of GNEM globally, and to accelerate development of a therapy for GNEM. They have recently created a brochure to further this cause.  Please visit their site.

Turkish GNE Myopathy Group
Many GNEM patients live in Turkey.  They have recently established a Facebook group. GNEM patients living in Turkey are encouraged to join this group..

Recent  GNE MyopathyNewsletters
1) Neuromuscular Disease Foundation, Summer Edition
2. GNEM-DMP  Eight Edition

The National Institutes of Health   NIH National Human Genome Research Institute
ManNac Trial Phase 3. Please monitor  this site for any updates and for when the trial will be recruiting patients. ManNac Trial Site.

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