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Home
About NDF
History & Mission
Organizational Structure
Who We Are
Board & Staff
Scientific Advisory Committee
Certified Patient Advocates
NDF Ambassadors
NDF United Youth
NDF's Road to Gene Therapy
FAQs
Programs & Financials
Programs
Clinical Research
Patient & Carrier Advocacy
Awareness & Education
Emotional Wellness
Financials
Special Donors
Sponsors & Partners
For Patients
FAQs
Recently Diagnosed?
Assistive Devices
Clinics
Wellness
Multidisciplinary Clinic
List of Physicians
Genetic Screening Information
NDF GNE Myopathy Registry Form
Patient Stories
Videos
For Physicians & Scientists
Resources for Physicians
Diagnostic Aids & Current Trials
Resources for Scientists
2021 Awarded Grants
Grants
Information, Updates and Studies
Scientific Studies
News & Events
Press & Media Links
Upcoming Events
Webinars
2020 GNEM Speaker Series
Podcasts
Photo Gallery
Past Events
Newsletter And Other Links
News Archive
Covid-19 Resource Page
Get Involved
Donate
Double Your Donation
Join Us
Ways to Fundraise
NDF Newsletter
Board Members Portal
CPA Portal
SAC Portal
Donation
Resources for Physicians
Diagnostic Aids & Current Trials
Resources for Scientists
2021 Awarded Grants
Grants
Information, Updates and Studies
Scientific Studies
Summary of NDF sponsored studies in 2020
Lessons learned from expanded reproductive carrier screening in self-reported Ashkenazi, Sephardi, and Mizrahi Jewish patients
Efficacy of ManNAc in Subjects with GNE Myopathy
GNE Myopathy
GNE myopathy: New name and new mutation nomenclature
Mutation Update for GNE Gene Variants Associated with GNE Myopathy
Sialylation of Thomsen-Friendenreich antigen is a noninvasive blood-based biomarker for GNE myopathy
Nemunaitis et al Hum Gene Ther 2011
Nishino et al JNNP 2015
Malicdan et al, Nature Med 2009
Xu et al.MGM 2017 ManNAc Ph1
Carrillo et al Neurotherapeutics 2018
GNE Phenotype Variability 2019
GNE Phase 3 Neurology 2019
Huizing et al Neuromusc Disord 2014