Sharon Hesterlee's Blog (28)

Without Endings There Are No Beginnings…

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…


Added by Sharon Hesterlee on January 5, 2015 at 1:30pm — 1 Comment

Now is the time for strength.

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.…


Added by Sharon Hesterlee on December 18, 2014 at 3:02pm — No Comments

No Exon Left Behind

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.…


Added by Sharon Hesterlee on July 21, 2014 at 10:15am — 6 Comments

New Duchenne Therapies in Development

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…


Added by Sharon Hesterlee on April 24, 2014 at 12:30pm — 3 Comments

PPMD Awards $100,000 in Exploratory Grants

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…


Added by Sharon Hesterlee on March 25, 2014 at 11:42am — 2 Comments

Getting There Faster

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…


Added by Sharon Hesterlee on November 26, 2013 at 3:45pm — 4 Comments

Who Owns Your Clinical Trial Data?

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…


Added by Sharon Hesterlee on October 29, 2013 at 11:00am — No Comments

World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

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…


Added by Sharon Hesterlee on October 4, 2013 at 9:30am — No Comments

DIA Oligonucleotide Conference, Neuromuscular Session Summary

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…


Added by Sharon Hesterlee on September 27, 2013 at 2:26pm — 1 Comment

GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen

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 (…


Added by Sharon Hesterlee on September 20, 2013 at 11:00am — 11 Comments

Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

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…


Added by Sharon Hesterlee on April 9, 2013 at 9:00pm — 5 Comments

A Strong Start to 2013

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…


Added by Sharon Hesterlee on February 4, 2013 at 2:30pm — No Comments

Sarepta Summary of Outstanding Questions from PPMD Webinar

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…


Added by Sharon Hesterlee on January 17, 2013 at 12:00pm — No Comments

Duchenne Drug Development Round Table

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…


Added by Sharon Hesterlee on November 15, 2012 at 10:00am — 5 Comments

Better, Faster, Now!

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…


Added by Sharon Hesterlee on October 11, 2012 at 11:00am — 4 Comments

Positive Phase 1 trial results for SMT C1100 in Duchenne (Summit PLC)

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


Added by Sharon Hesterlee on October 10, 2012 at 10:30am — 13 Comments

Parent Project Muscular Dystrophy awards $50,000 to Dr. Toshifumi Yokota for multi-exon skipping project

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…


Added by Sharon Hesterlee on September 27, 2012 at 2:30pm — No Comments

Interview with Chris Garabedian, President and CEO of Sarepta Therapeutics

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…


Added by Sharon Hesterlee on August 31, 2012 at 10:30am — 2 Comments

Increasing stem cell survival in Duchenne therapy

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…


Added by Sharon Hesterlee on August 29, 2012 at 2:00pm — 6 Comments

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