Added by John D. Porter, PhD on December 21, 2015 at 11:03am — No Comments
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). …
ContinueAdded by John D. Porter, PhD on December 17, 2015 at 4:00pm — No Comments
Last week I participated in a briefing for Congressional staff in honor of the 10th anniversary of the founding of the Critical Path Institute, our partner in the Duchenne Regulatory…
ContinueAdded by John D. Porter, PhD on October 27, 2015 at 10:50am — 4 Comments
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…
ContinueAdded by John D. Porter, PhD on October 16, 2015 at 1:07pm — No Comments
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…
Added by John D. Porter, PhD on October 16, 2015 at 11:30am — No Comments
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…
ContinueAdded by John D. Porter, PhD on September 16, 2015 at 11:00am — No Comments
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…
ContinueAdded by John D. Porter, PhD on August 3, 2015 at 9:00am — No Comments
Yesterday, Parent Project Muscular Dystrophy and Duchenne Parent Project-Netherlands (DPP) co-hosted the first ever conference on assistive technology for Duchenne muscular dystrophy. There were twenty-five participants in the workshop, from six countries, representing academia (both researchers and engineers), companies, clinical practice, and patient advocacy. PPMD was represented by Pat Furlong, Annie Kennedy, and myself. Elizabeth Vroom (DPP-Netherlands) set the tone for the meeting…
Added by John D. Porter, PhD on April 28, 2015 at 10:00am — No Comments
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…
ContinueAdded by John D. Porter, PhD on April 21, 2015 at 10:00am — No Comments
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…
ContinueAdded by John D. Porter, PhD on March 21, 2015 at 10:30am — 6 Comments
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…
ContinueAdded by John D. Porter, PhD on February 12, 2015 at 12:00pm — 6 Comments
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…
ContinueAdded by John D. Porter, PhD on January 12, 2015 at 10:00am — 1 Comment
© 2021 Created by PPMD.
Powered by
Badges | Report an Issue | Privacy Policy | Terms of Service