The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.
"Five years ago, this meeting couldn’t have happened," said Jane Larkindale, MDA's vice president of research. "We couldn’t have filled two-and-a-half days with the kinds of presentations we have now. The agenda is packed with important talks discussing everything from early-stage therapeutic targets through to clinical trial results, along with sessions discussing the development of new tools that will allow us to conduct trials more effectively."
The conference is aimed at professionals and is not open to the public. However, blogs and reports will be available to all on the meeting website, both during and after the proceedings.
Nearly 500 attendees from academic laboratories, clinics and industry are expected, with more than 60 platform presentations and more than 200 poster presentations planned.
The goal, said Larkindale, is to “identify barriers to therapeutic development and how to overcome those barriers.”
Conference co-chairs are C. Frank Bennett, CEO of the biomedical company Isis Pharmaceuticals, and Eric Hoffman, director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C.
Conference presentations have been organized into broad themes relating to therapy development.
Targets: These presentations will focus on identifying molecular targets at which to aim therapies in different disorders, such as amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), facioscapulohumeral muscular dystrophy (FSHD), myotonic muscular dystrophy (MMD) and spinal muscular atrophy (SMA).
Genetic modifiers: Presentations will explore naturally occurring gene variations that modify the course of diseases and can help explain an individual's disease course, as well as provide targets for therapeutic development, in disorders such as ALS, Becker muscular dystrophy (BMD), Duchenne muscular dystrophy (DMD), SMA and MMD.
Therapeutic modalities: Presentations will focus on different types of compounds that have the potential to be developed into therapies, such as stem cells, proteins, small molecules and antisense, with particular application to ALS, one form of congenital muscular dystrophy (CMD), DMD and FSHD.
Biomarkers: Presentations will discuss the identification and use of biological indicators, including imaging studies, which may reflect the progress of a disease or its response to treatment, with particular application to ALS and DMD.
Animal models: Discussions will center on research animals that replicate the characteristics of various human neuromuscular diseases and how they can be used to study these diseases, with particular application to ALS, centronuclear myopathy (CNM), DMD, Emery-Dreifuss muscular dystrophy (EDMD) and SMA.
Use of animal models in drug development: Presenters will explore how well animal models of human disorders can be used to develop drugs, with particular application to ALS, DMD FSHD, myotubular myopathy (MTM) and SMA.
Preclinical work for trial design and regulation: Designing laboratory studies that can serve as the foundation for human trials of investigational drugs will be focus of these talks.
Clinical trials: These presentations will include updates on trials of tirasemtiv in ALS; drisapersen and eteplirsen in DMD; cardiac treatments for DMD; RG2833 for Friedreich's ataxia (FA); and ISIS-SMNRx for SMA.
The latest and greatest: Discussions will include late-breaking research reports, with particular application to CNM, DMD, FA and mitochondrial myopathies.
Resources for drug development: Topics to be covered include working with the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS), tissue banks, stem cells, databases, and computer chips, with particular application to ALS.
To learn more and to read blogs and reports during and after the meeting, see the MDA Scientific Conference home page.
This conference is the third in MDA's conference series, which features scientific (research-oriented) conferences in odd-numbered years, and clinical (care-oriented) conferences in even-numbered years.