Therapy development for neuromuscular diseases: translating hope into promise.

The Second Muscular Dystrophy Association Scientific Meeting was held on 21-24 April 2013 in Washington (DC, USA). The meeting provided an opportunity for research scientists, clinicians, government agencies and industry experts to highlight and discuss different aspects of therapy development for neuromuscular diseases, including novel targets, biomarkers, therapeutic approaches, animal models and clinical trials. With 500 participants, 66 presentations and 200 abstracts, the 3-day conference has become a central focus for scientists interested in translational research and moving potential therapies forward from the bench to the bedside. Key issues covered by the meeting included the need to identify new drugs to treat patients with neuromuscular diseases and the importance of establishing collaborations between government, academic and industry sectors toward an efficient and rigorous translational path for neuromuscular diseases.