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…

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…

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…

The promise of gene therapy continues to build momentum, in Duchenne and also in other conditions. Much has been learned about the immunological and delivery challenges of the approach since gene therapy’s early attempts in the 1990s.

In Duchenne, some of the early work in gene therapy ended with immune system reactions to the new dystrophin that was expressed by the transfected gene. But this was not…

A couple of months ago we posted a write up about some of the efforts PPMD is undertaking to help improve…

There has been a flurry of press recently on gene therapy in Duchenne muscular dystrophy and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.

An Expert…

The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duchenne therapeutic in development now in some way. In FY2016, the NIH…

Gene therapy.

Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene…

It seems that everyone is looking for the newest molecule, the newest therapy, the newest technique that will halt the progression of Duchenne. This is good and we should continue on this journey. But none of these novel treatments will get to patients without first going through a rigorous…

This year’s already action-packed Advocacy Conference, February 21-23 welcomes a special Gene Therapy Forum on Monday, February…