Funded By:
July 30, 2007
Rehabilitation Research and Training Center in Neuromuscular Diseases
University of California, Davis
Sacramento, CA
On July 30, 2007 approximately 100 boys and family members of boys with Duchenne muscular dystrophy (DMD) attended a conference put on by the Rehabilitation Research and Training Center in Neuromuscular Diseases (RRTC NMD) at the University of California at Davis. Faculty members of the RRTC NMD presented the latest in research on DMD. Dr. Craig M. McDonald, Director of the RRTC, welcomed everyone and introduced the participants. The slide presentations of each of the speakers are available by clicking on the links below. It is necessary to have Adobe Reader to open them. Adobe Reader is available for free download by clicking here.
“New Frontier for Treatment of Duchenne Muscular Dystrophy: Pharmacotherapies, Gene Transfer and Stem Cell Treatments” by Jay Han, MD was the first presentation. This presentation discussed various therapeutic approaches for DMD, pharmacologic therapy, gene therapy and cell therapy.
“Role of PTC 124 for the Treatment of DMD” was the topic of Leone Atkinson, MD, PhD., representing PTC Therapeutics, the developers of the drug PTC 124. PTC 124 is a drug currently in development and being tested in clinical trials for the treatment of DMD. This presentation presented the background of the basis for the use of PTC 124 and a review of the clinical trials that have been conducted and those currently recruiting participants.
“Exon Skipping and the Role of Morpholinos for Treatment of DMD” by Craig McDonald, MD. Dr. McDonald presented slides that were provided courtesy of Qi Long Lu. Toshifumi Lokota, and Terry Partridge of the MRC, Steve Wilton of ANRI Perth , Glen Morris of NWRI Wales , and Shin'ichi Takeda of the National Institute of Neuroscience , Tokyo . This is a fairly technical presentation of what exons are and how antisense oligonucleotides (morpholinos, AOS) are being used experimentally to “skip” faulty exons and allow production of modified dystrophin molecules.
“Translation of Promising Therapeutic Compounds to Clinical Trials in DMD” by Andrew Skalsky, MD. This presentation gave an overview of the process of drug development from the initial stages through the various steps of clinical trials that lead up to the final approval of a new drug or a new use of an existing drug by the Food and Drug Administration.
“Getting Better Information: Improving Health and Management of DMD” by Craig McDonald, MD. This presentation has several areas of focus. An overview of the goals of the RRTC NMD is followed by a presentation of the studies currently being conducted to develop an accurate natural history of DMD. The next part covers issues of living with DMD that have been identified as needing study by a workgroup at NIH and the obstacles, challenges and opportunities to accomplishing this work. This is followed by information on a series of panels organized by the CDC and the RRTC NMD to evaluate the status of clinical care considerations for DMD. The concluding section presents future goals and the infrastructure needed to accomplish these goals.
