John D. Porter, PhD's Blog (12)

A New Dog Model for Drug Development in Duchenne

Photograph: Chris Kench/ via The Guardian



Added by John D. Porter, PhD on December 21, 2015 at 11:03am — No Comments

Dystrophin Mutations Affect Not Only Existing Muscle Fibers, but Satellite Cells As Well

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).  …


Added by John D. Porter, PhD on December 17, 2015 at 4:00pm — No Comments

A Paradigm Shift in Drug Discovery

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…


Added by John D. Porter, PhD on October 27, 2015 at 10:50am — 4 Comments

Annual Meeting: Patient-Centered Outcomes Research Institute (PCORI)

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…


Added by John D. Porter, PhD on October 16, 2015 at 1:07pm — No Comments

Preclinical Studies with Simvastatin in the Mdx Mouse Model of Duchenne

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

The Power is in Your Hands: Duchenne Regulatory Science Consortium

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…


Added by John D. Porter, PhD on September 16, 2015 at 11:00am — No Comments

Different Together: PPMD Announces the Duchenne Regulatory Science Consortium

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…


Added by John D. Porter, PhD on August 3, 2015 at 9:00am — No Comments

Let's Get Them Moving Workshop: Our First Steps Toward That Goal

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

Santhera Releases Positive Data from Phase III Clinical Trial of Idebenone (Raxone®/Catena®) in Duchenne

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…


Added by John D. Porter, PhD on April 21, 2015 at 10:00am — No Comments

Recapping the FDA-NIH Dystrophin Methodology Workshop

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…


Added by John D. Porter, PhD on March 21, 2015 at 10:30am — 6 Comments

Your Action Plan for the Muscular Dystrophies

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…


Added by John D. Porter, PhD on February 12, 2015 at 12:00pm — 6 Comments

Where the Long, Winding Path has Led

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…


Added by John D. Porter, PhD on January 12, 2015 at 10:00am — 1 Comment

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