blog

Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases

Tue, 31 Jan 2012 09:27:04 -0600

With 14 approved programs in total, TRND has progressed two into clinical development.

For the past two years NIH has been trying to double its funds for developing orphan drugs. But the FY 2012 budget offered more of the same, as in the same $24 million of annual funding set aside for NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program since its creation in 2009. The institute had sought $50 million for TRND this year and last.

Genetic Engineering & Biotechnology News, January 31, 2012 (Alex Philippidis)

AVI Enters Into Collaborations for the Development of Two Additional Exon-Skipping Products for Duchenne Muscular Dystrophy (DMD)

Thu, 17 Nov 2011 12:20:37 -0600

Collaborations Represent Key Inflection Point in AVI's Progress in Developing Treatments for Broader DMD Population

BOTHELL, WA, Nov 15, 2011 (MARKETWIRE via COMTEX) --

KELLY GILFILLAN: Outside the Brentwood Bubble : Local family helps raise money for Duchenne MD

Tue, 04 Oct 2011 21:00:00 -0500

Local Family Helps Raise Money for Duchenne MD

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy

Tue, 06 Sep 2011 09:00:00 -0500

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy

http://prosensa.eu/press-release/results-prosensa%E2%80%99s-extended-phase-iii-exon-skipping-trial-duchenne-muscular-dystrophy-

ReveraGen/TRND Partnership

Tue, 06 Sep 2011 08:52:00 -0500

The ReveraGen/TRND Press Release

Department of Defense Duchenne Muscular Dystrophy Research Program

Tue, 06 Sep 2011 08:40:00 -0500

Department of Defense Duchenne Muscular Dystrophy Research Program

http://cdmrp.army.mil/pubs/press/2011/11dmdrppreann.shtml

First Treatment for Muscular Dystrophy in Sight

Sun, 01 Mar 2009 22:59:00 -0600

First Treatment for Muscular Dystrophy in Sight: Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy

Researchers from Children’s National Medical Center and colleagues in Tokyo publish results, video of first successful trial in dogs with Duchenne muscular dystrophy. Click here to read more.

Congress Appropriates Duchenne Muscular Dystrophy Translational Research Funding Through the Department of Defense

Tue, 01 Jan 2008 10:21:00 -0600

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.

Promising Dystrophy Drug Clears Early Test

Thu, 27 Dec 2007 10:25:00 -0600

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

FED announces financial support for the first international workshop

Wed, 29 Aug 2007 22:26:00 -0500

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.

Senate proposes Duchenne muscular dystrophy translational research

Sat, 25 Aug 2007 10:27:00 -0500

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.

Department of Defense approves $1.7 million of support

Sat, 18 Aug 2007 10:30:00 -0500

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.

The Program Project for Translational Research

Fri, 10 Aug 2007 10:31:00 -0500

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.

FED helps form the IDEW Group

Thu, 12 Jul 2007 10:17:00 -0500

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.

FED sponsors an international workshop

Mon, 15 Jan 2007 10:21:00 -0600

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.

FED funds large-scale production of multiple morpholinos

Fri, 15 Dec 2006 20:32:00 -0600

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.