Overview

Therapy Development for Neuromuscular Diseases: Translating Hope Into Promise

MDA’s 2013 Scientific Conference was held April 21-24, 2013, in Washington, D.C. The sold-old event, which attracted more than 500 participants from around the world, focused on therapeutic strategies for neuromuscular diseases, with sessions highlighting different aspects of therapy development. Some 70 platform presentations and 200 poster presentations were included in the two-and-a-half days, along with time for networking and discussion.

Capitalizing on MDA’s unique status as an umbrella organization covering more than 40 neuromuscular diseases, the tightly packed agenda focused on common research themes — rather than individual diseases — including novel drug targets, biomarkers, therapeutic modalities and new animal models. A “late-breaking” session provided a glimpse of some of the most up-to-date research in the field.

The unprecedented number of experimental therapies in clinical trials, the changing landscape of orphan drug research and development, and the many opportunities for information-sharing and collaboration among scientific professionals from many disciplines made for a lively and exciting meeting.

To read more about MDA’s 2013 Scientific Conference, see ‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research.