Over the past several years significant progress has been made in the research area of dystroglcyanopathy. These advances include: the identification of various new genes bringing new insight into the pathway(s) involved in the glycosylation of alpha-dystroglycan; AAV-mediated gene therapy in FKRP animal models continues to show promising results; and new directions are being pursued for the functional glycosylation of alpha-DG. Although the advancements being made have proven beneficial a complete understanding of the system remains unknown and hurdles still remain in our attempts to treat the disease.
The focus of the 2015, 4th International Workshop for Glycosylation Defects in Muscular Dystrophy will be to address the potential for identifying drugs and therapies to upregulate functional glycosylation as well as AAV mediated gene therapy for FKRP-related diseases and the barriers being faced. As with the previous workshop, advances in clinical management and development of endpoint markers will provide a major 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.
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 with a focus on new developments in gene therapy, drug development and patient care.
|All programs will be held at:
Fairfield Inn & Suites Charlotte Uptown
|Day One–Thursday, April 16, 2015:
||Day Two–Friday, April 17, 2015:
|Details for Attendees||Sponsor and Host Information|