It’s New Year’s Day 2015 and I think Janus, the two-headed Roman God who looks forward and backwards at the same time, is a fitting representation of my life right now. I need to tell you all that I have had to make a difficult decision this year—at the end of this month I will be winding up my time at PPMD while simultaneously starting mid-month with the Myotonic Dystrophy…

We’ve been working toward this moment for years. And the time for action is now.

When I started with PPMD in 2009, Pat Furlong and I talked a lot about what it would take to end Duchenne. We knew it wouldn’t be one thing, and we knew it wouldn’t be easy. We believed that only a combination of therapies could address the entire disease within the body.…

I'm often asked by parents to explain exon skipping. In fact, at last month's 20th Annual Connect Conference, after we announced a grant awarded to Dr. Judith van Deutekom, parents wanted to know what it is we are actually funding.…

As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…

PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has been tested in other disorders and has been shown to correct some…

PPMD’s research strategy has long had three distinct facets—to develop treatments Better, Faster, and Now. In the past, I have talked about how we have teamed up with TREAT-NMD to use its panel of experts on Duchenne and drug development to review projects with the goal of putting…

When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school, waiting rooms, endless syringes, and painful biopsies are…

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.

Next Generation Exon-Skipping

Several laboratories presented data on ways…

At a Washington meeting last week on oligonucleotide-based therapeutics  co-sponsored by the Drug Information Association (DIA) and the Food and Drug Administration, several speakers addressed recent developments in the use of antisense oligonucleotides for Duchenne muscular dystrophy.  Allison Durham of FaegreBD captured the highlights of those talks for PPMD.

Update on Sarepta’s AVI-4658 in Exon 51 Skipping Amenable Duchenne Muscular Dystrophy…

UPDATE AS OF 10/4/13: We received the following Q&A from GSK today, addressing many of the community's questions. Click here.

Today we learned that GSK’s phase III study of the exon 51 skipping drug drisapersen failed to show a statistically significant improvement in the six-minute walk test compared to placebo (…

This spring brings news of progress to the Duchenne community as Sarepta engages in talks with the Food and Drug Association (FDA) to determine if they agency will review an application for accelerated approval for the exon 51 skipping drug eteplirsen and releases its 74 month data…

Thanks to everyone who contributed so generously to PPMD’s holiday appeal we have been able to fund three new projects designed to speed the progress of the clinical trials underway in Duchenne. As many of you know, we have a…

On October 23, 2012, PPMD hosted a webinar with Sarepta to discuss of the 48 Week dystrophin and 6-minute walk test (6MWT) data from the Phase IIb extension study in Duchenne as well as next steps for the eteplirsen program. Chris Garabedian, President and CEO of Sarepta…

There are a lot of frustrations in the Duchenne community—one of the most common themes I’ve heard is “why isn’t all this research and drug development coordinated better?” The problem, of course, is that there are a lot of companies and funding groups trying to help at once—sometimes it may feel like too many cooks in the kitchen, but in the long run, I think this is a much…

These are the words that best describe PPMD’s research plan.

1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
2. We apply funding strategically to increase the speed of testing new drugs.
3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.
   
   
   
   

Everything we do goes through this filter to make sure that we are achieving one of these…

Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit…

We are so happy to support the work of Dr. Toshifumi Yokota at the University of Alberta, in Canada. Exon skipping looks promising as a viable therapeutic for Duchenne. Dr. Yokota is taking…

We are all acutely aware of the need for an effective treatment for Duchenne. Finding a way for children and young men affected by Duchenne to lead longer, healthier lives is at the heart of all of our efforts as families’ fight this devastating disease.

This week PPMD received an…

One approach to improving muscle strength in Duchenne is to replace unhealthy muscle cells that lack dystrophin with donor stem cells that have normal levels of dystrophin. These cells might come from a relative unaffected by Duchenne or even a person with Duchenne’s own cell, after dystrophin is restored though a technique like gene therapy.

Although we’ve…

As Sarepta (formely AVI) announces promising results from its phase II interim data and GSK follows up its promising phase…