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Third International Workshop for Glycosylation Defects in Muscular Dystrophies

In recent years, significant progress has been made in the research area of dystroglcyanopathy. These advances include: the identification of functional sugar units on α-DG; the proposal of new pathways involved in functional glycosylation and laminin binding; new data available for AAV-mediated gene therapy in FKRP animal models; and compound library screening in several labs have resulted in hits with the potential for upregulating the functional glycosylation of α-DG or other proteins, thus providing potential drugs for dystroglycanopathies. All of these data are relatively preliminary and each approach still faces formidable hurdles.

The focus of the 2013 Third International Workshop for Glycosylation Defects in Muscular Dystrophy will be to address the potential for identifying drugs to upregulate functional glycosylation by screening as well as AAV mediated gene therapy for FKRP-related diseases and the barriers being faced. Furthermore, advances in clinical management and development of endpoint markers will provide a topic of discussion.

The workshop will bring together scientists and clinicians with active research in these areas to discuss the significance of the updated experimental data and facilitate potential collaboration to possibly identify new approaches to addressing disease treatment.

Workshop Information (Two-Day Sessions)
All programs will be held at:

Omni Charlotte Hotel
132 E. Trade St.
Charlotte, NC 28202

View program overview (PDF)

Day One–Thursday, April 18, 2013:
  • Registration and breakfast
  • Morning session(s)
  • Lunch
  • Afternoon session(s)
  • Banquet
Day Two–Friday, April 19, 2013:
  • Registration and breakfast
  • Morning session(s)
  • Lunch and closing remarks
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