The 4th International Workshop for Glycosylation Defects in Muscular Dystrophy took place in Charlotte April 16-17, 2015.
Twenty-one speakers, representing four countries and 10 states in the US, presented their latest research from the perspectives of top scientists and clinicians in the field of muscular dystrophy. The spectrum of speaker talks highlighted the translational successes resulting from these experts collaborating to advance therapies for patients with various forms of muscular dystrophy. Scientists presented the progress made in understanding the mechanisms of the disease as well as various experimental therapies, including gene therapies. Clinicians presented the challenges of counseling patients with gene profiles due to the varied clinical presentation of the disease even among those with shared gene mutations. They also discussed the challenges of designing and conducting clinical trials due to varied clinical presentations. Many clinicians acted as patient advocates to remind the audience that the functional outcomes, such as the ability to feed or clothe oneself, are paramount for quality of life. Finally, the results of collaboration and funding were demonstrated by the success of moving therapies from the laboratory into the clinic in a strategic and targeted way.
The Workshop provides a unique opportunity to have a broad spectrum of experts in the same room where they respectfully challenged each other to think outside the box in order to advance research in this field. Speakers expressed hope because significant milestones were reached and progress has been made since the previous Workshop. This hope continues as we look forward to the milestones and successes that are sure to come before the next Workshop in 2017.
The audience comprised clinicians, laboratory scientists, patient advocates, funding agencies, industry representatives, and research support staff, united in the mission to get an effective therapy to patients living with muscular dystrophy.
The event was sponsored by Carolinas HealthCare Foundation, The McColl and Lockwood families and the ‘Riding 4 Research’ fundraiser. The McColl-Lockwood Laboratory for Muscular Dystrophy Research served as the event host.