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…
Added by Abby Bronson on December 12, 2017 at 9:00am — No Comments
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…Continue
Added by Abby Bronson on November 30, 2017 at 6:30pm — No Comments
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,…Continue
Added by Abby Bronson on October 24, 2017 at 3:00pm — No Comments
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…Continue
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…Continue
Added by Abby Bronson on October 2, 2017 at 1:30pm — No Comments
As you know by now, September is Duchenne Action Month. And while the idea behind this campaign is to encourage our community to take…Continue
Added by Abby Bronson on September 21, 2017 at 12:00pm — No Comments
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…Continue
Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments
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…
Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments
Added by Abby Bronson on July 31, 2017 at 10:46am — No Comments
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…Continue
Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments
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…Continue
Added by Abby Bronson on May 16, 2017 at 3:00pm — No Comments
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…Continue
Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments
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…Continue
Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments
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…Continue
Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments
Help us raise $250,000 by 12/31!…
Added by Abby Bronson on December 20, 2016 at 12:00pm — No Comments
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…Continue
Added by Abby Bronson on December 8, 2016 at 3:30pm — No Comments
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…Continue
Added by Abby Bronson on October 18, 2016 at 1:00pm — No Comments
Added by Abby Bronson on September 8, 2016 at 9:43am — No Comments
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…Continue
Added by Abby Bronson on August 12, 2016 at 10:09am — No Comments
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:
Added by Abby Bronson on June 16, 2016 at 11:04am — No Comments