January, 2008 - Congress Appropriates Duchenne Muscular Dystrophy Translational Research Funding Through the Department of Defense.

The Fiscal Year 2008 Department of Defense budget includes key funding for muscular dystrophy research in the amount of $9.2 million. These federal appropriations are part of a newly-created National Program that will be used to promote therapeutic approaches such as exon skipping for Duchenne Muscular Dystrophy. This is the largest federal appropriation for clinical translational research in Duchenne dystrophy to date.

December 27, 2007 - Skipping toward Personalized Molecular Medicine - Dr. Eric Hoffman Op-Ed in the New England Journal of Medicine

December 27, 2007 - Promising Dystrophy Drug Clears Early Test http://www.nytimes.com/2007/12/27/health/27drug.html?_r=1&fta=y&oref=slogin 

August 29, 2007 - FED announces financial support for the first international workshop on pre-clinical methods in mouse drug screening trials in the muscular dystrophies.

The National Institutes of Health Wellstone network has sponsored workshops on key current topics in translational and clinical research in the muscular dystrophies, and recently announced the award of a workshop on standardization of methods for drug testing in mouse models of muscular dystrophy. As more is known about the causes of the muscular dystrophies, and more mouse models become available, it becomes increasingly important to test multiple therapeutic approaches in the mouse models, as a 'proof-of-principal' prior to the start of expensive and time consuming human clinical trials. However, methods used to carry out mouse pre-clinical trials vary from laboratory to laboratory, making it difficult to compare results, and judge when best to move to human trials. This workshop, organized by Kanneboyina Nagaraju of Children's National Medical Center, will bring together top investigators from around the world, with the goal of reaching a consensus to most rapidly move pre-clinical testing forward. FED is proud to co-sponsor this workshop with the National Institutes of Health.

August 25, 2007 - Senate proposes Duchenne muscular dystrophy translational research earmarks within the Department of Defense.

The Department of Defense budget currently under negotiation in the House of Representatives includes key funding for muscular dystrophy research, with approximately $10 million proposed by the Senate. This would provide the largest federal earmark for clinical translational research in Duchenne dystrophy to date.

August 18, 2007 - Department of Defense approves $1.7 million of support for clinical trial infrastructure to the Cooperative International Neuromuscular Research Group (CINRG).

The Cooperative International Neuromuscular Research Group is the largest clinical trial network for muscular dystrophy world-wide, with 22 sites in 11 countries. The Department of Defense is supporting clinical trials in Duchenne dystrophy, with key funding to two top-10 ranked Children's Hospitals: Children's Hospital of Pittsburgh, and Children's National Medical Center in Washington, DC.

August 10, 2007 - The Program Project for Translational Research in Muscular Dystrophy receives a $1.6 million boost from FY07 Department of Defense funding.

A consortium of top laboratories working on translational research in Duchenne dystrophy has received a supplement to their FY2005 program project to expand their successful work in exon skipping research (anti-sense oligonucleotides). Laboratories participating in the DoD Program Project include Carrie Miceli of UCLA, Qi Lu of Carolinas Medical Institute, Terence Partridge of Children's National Medical Center, Paul Morcos of GeneTools Inc, Toshifumi Yokota of Children's National Medical Center, and Kanneboyina Nagaraju of Children's National Medical Center. Key collaborators include Shin'ichi Takeda of the National Institutes of Neuroscience in Japan.

July 12, 2007 - FED helps form the IDEW Group (International Duchenne Exon-skipping Working Group) to push forward clinical trials in exon-skipping methods.

The International Duchenne Exon-shipping Working Group (IDEW group) was formalized as an outcome of successful pre-clinical research, and proposed clinical trials resulting from FED, Department of Defense, and Japanese Government funding to a consortium of top laboratories. The Japanese government has provided a $800,000/yr competitive research grant to Shin'ichi Takeda MD for approaches using morpholino cocktails and systemic delivery. FED provided seed money to initiate a murine pre-clinical drug screening core in Washington DC, and funding to Carrie Miceli and Stan Nelson at UCLA to develop high throughput drug screening protocols to enhance potency of exon-skipping delivery. FED also supported initial morpholino production costs for systemic delivery and toxicity studies. The US Department of Defense has provided a $3.7 million program project to the above investigators, as well as Dr. Qi Lu for work on systemic dose/response and efficacy studies in mouse models. The IDEW group coordinates their research with conference calls and meetings, and through international workshops with other researchers working on exon-skipping.

May 8, 2007 - Dr. Eric Hoffman's comments before Queen Elizabeth II during her recent visit to Children's National Medical Center

January 15, 2007 - FED sponsors an international workshop on exon skipping approaches in Duchenne dystrophy.

A workshop was organized by Eric Hoffman at Children's National Medical Center to discuss progress towards human clinical trials in exon-skipping approaches in Duchenne dystrophy. Judith Vandeutekom and Prosensa described their success of local muscle injections in patient with DMD. AVI and Dr. Francesco Muntoni described their progress towards a morpholino clinical trial. Drs. Hoffman, Partridge, and Takeda showed their success in systemic delivery in naturally-occurring dogs with Duchenne dystrophy. Approximately 40 invitees attended the workshop, including representatives from NIH, FED, and FDA.

December 15, 2006 - FED funds large-scale production of multiple morpholinos for systemic delivery trials.

FED approved $450,000 in morpholino production costs to Gene Tools, LLC of Oregon, for large-scale, high-grade material used for systemic delivery pre-clinical trials of naturally occurring dogs with Duchenne dystrophy. The systemic delivery trials were carried out by the National Institutes of Neuroscience in Japan.