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Don't miss our all the news in our September News Letter



Please Join us in Great Neck, New York on Sunday, January 11, 2015 to learn about the current scientific research in GNEM and what you can do to help! Please RSVP by January 2, 2015

Questions? Please contact Carol Gelbard at


11:00-12:00 Brunch

12:00-12:15 Welcome and Opening Remarks
Carolyn Yashari Becher, Esq.
Neuromuscular Disease Foundation
Los Angeles, CA

12:15-12:45 Overview of Current Research in HIBM
Zohar Argov, MD
Hadassah-Hebrew University Hospital
Jerusalem, Israel

12:45-1:30 Recent Results of the Sialic Acid Study
Heather Lau, MD
New York University
New York, NY

1:30-2:15 Recent Results of the ManNAc Study & NIH Natural History Study
Nuria Carrillo-Carrasco, MD
National Institutes of Health
Bethesda, MD

2:30-3:00 Why is the GNEM-DMP (Natural History and Registry) Important?
Alison M. Skrinar, PH.D.
Ultragenyx Pharmaceutical Inc.
Novato, CA

3:00-3:45 Genetic Screening for HIBM
Gidon Akier, MD
Icahn School of Medicine at Mount Sinal and SHORE
(Sephardic Heath Organization for Referral and Education)
New York, NY

3:45-4:00 Wrap up of Learnings
John Hakimi, PhD
Sage BioConsulting Inc.
Scarsdale, NY

Sponsored by the Neuromuscular Disease Foundation

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.


GNE Myopathy is also known as: HIBM,  Nonaka  Myopathy, Distal Myopathy With Rimmed Vacuoles,  Inclusion Body Myopathy 2,  IBM 2, Quadriceps Sparing Myopathy

View our report by clicking here

NDF is a registered 501(c)(3) nonprofit organization (EIN: 06-1789643)