Photograph: Chris Kench/www.chriskenchphotography.com via The Guardian

Some…

A recent paper by Michael Rudnicki’s group at Ottawa Hospital Research Institute and the University of Ottawa has provided new insights into what we view as the critical targets for therapies in Duchenne (Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division. Dumont et al., Nature Medicine 21 (12): 1455-63, 2015).  …

Last week I participated in a briefing for Congressional staff in honor of the 10^th anniversary of the founding of the Critical Path Institute, our partner in the Duchenne Regulatory…

The first annual meeting of PCORI was held in Washington, DC on October 6-8.  1,100 researchers, patients, caregivers, health system and industry representatives, and others were present to hear updates on patient-centered comparative clinical effectiveness research.  PCORnet is a “network of networks” that leverages databases and experience across diseases…

Stan Froehner’s group at the University of Washington recently reported preclinical efficacy data on the approved drug, Simvastatin, in the mdx mouse model of Duchenne (see PPMD's blog from the last day of the World Muscle…

About a month ago, I wrote to you to announce the launch of a new partnership between PPMD and the Critical Path Institute (C-Path) called the Duchenne Regulatory Sciences Consortium or D-RSC (see “Different Together: PPMD Announces the…

Signal to Noise

This is a measure used in science and engineering to compare the level of a desired signal (a measured outcome) to the background noise. In a way, this is the question of what information carries weight/is meaningful and what information clouds the issue.

While this sounds really complicated, we have witnessed…

Today, Santhera has published the results in the highly prestigious journal, The Lancet, from their successful clinical trial of Raxone®/Catena® . They will present these results later this week, at the annual meeting of the American Academy of Neurology. First, idebenone was safe and well tolerated. Importantly, treatment of study…

I always felt a strong connection to the Duchenne community while working at the NIH. As you know, that connection and the incredible work of organizations like PPMD inspired me to jump full-time into the fight to end Duchenne.

Yesterday’s FDA and NIH dystrophin methodology workshop really cemented that decision for me. Never have I been more proud to…

During my time at the National Institutes of Health (NIH) one of my key roles was serving as Executive Secretary for the Muscular Dystrophy Coordinating Committee (the MDCC). To help coordinate activities across the NIH and with activities of other Federal health agencies, the MDCC has undertaken two major planning efforts. Before leaving NIH…

I sit here in my home office on a snowy January day in Maryland, and I think about where I am at now in life and how I got here. As a naïve, newly minted Ph.D. in 1980, it wasn’t inevitable, or even predictable, that I would ultimately join all of you in the fight to end Duchenne. But, I’m really glad that I’m here and I’ll tell you a bit about the path along…