We’ve entered a new chapter for families with Duchenne, and 2017 has been a year of incredible promise.

Yes, we’ve faced some setbacks, but we’ve also seen incredible successes, like recent breakthroughs in gene therapy. It’s essential that we keep…

Throughout the history of Duchenne, there have been moments that we recognize as inflection points, times when we saw a significant shift in the fight to end Duchenne, when the…

Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease.

But progress is being made and PPMD is proud of our role in changing the clinical trial process. Last week,…

On September 28 and 29, I had the privilege of attending an in-person Wellstone Center meeting that Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center hosted in Dallas, TX. The breadth and depth of what the Wellstone Centers have worked on over the years was apparent. I was struck by the number of therapies in the clinic…

This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including…

As you know by now, September is Duchenne Action Month. And while the idea behind this campaign is to encourage our community to take…

Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb, remarked on the FDA's approval of the first gene…

With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.

Earlier this month, PPMD hosted a webinar with Lee Sweeney, PhD (University…

PPMD, at the start of the year, made its biggest investment ever, $2.2M to…

An Overview of Your Impact in Action

We are less than a week away from PPMD’s Annual Connect Conference, where researchers and industry from around the world will provide…

On May 9, the Duchenne Regulatory Science Consortium (DRSC) held its Annual Meeting in Crystal City, VA. In the audience of about 40 people, there were industry and academic Consortium members, potential industry members, five FDA representatives, two NIH representatives, C-PATH representatives, and Buddy Cassidy, the DRSC…

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical…

An Overview of Your Impact in Action

PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.

PPMD Launches Long-Term…

Better. Faster. Now.

How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?

Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…

Help us raise $250,000 by 12/31!…

It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.  

Could CRISPR/Cas9 offer hope for treating Duchenne? 

As background, CRISPR/Cas9 is a…

When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in Duchenne history. There were three compounds in the clinic (drisapersen, ataluren, and eteplirsen aka Exondys 51) with more waiting in the wings to go into clinical trials, a drug…

Quercetin /ˈkwɜːrsᵻtᵻn/ is a flavonol found in many fruits, vegetables, leaves and grains. It can be used as an ingredient in supplements, beverages, or foods. Source:…

Last month, PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9. If you missed it live, check out the recording to learn more about this technology and what it…

You’ve seen the headlines and heard the name, now find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duchenne during three different opportunities that promise to provide an in-depth analysis:

• Gene Therapy and CRISPR Cas9: Similarities and differences, Tuesday June 28…