September 2013 Blog Posts (12)

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

Impact of Drisapersen Phase 3 Data on Prosensa Programs

Prosensa has provided the following update regarding the impact of the drisapersen phase 3 data on its programs: 

While we are disappointed and surprised with the unexpected outcome of the drisapersen phase 3 study, we remain very committed to drisapersen, the Duchenne program at…


Added by PPMD on September 24, 2013 at 11:30am — No Comments

Anybody In HT-100 Trial?


I would like to know if anybody is enrolled in HT-100 (Halofuginone ) trial?

Added by Moein on September 21, 2013 at 10:17am — No Comments

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

My Voice: TJ Clime, 9 years old

On September 28, 2013, families across the country will celebrate PPMD's Sixth Annual Coach To Cure MD. Coaches from nearly 600 universities and colleges around the country will wear a Coach To Cure MD patch to help raise awareness for Duchenne. Hundreds of high school coaches (and entire teams!) will also support this amazing event. TJ Clime and his family suited up and started supporting Coach To Cure MD weeks ago. This incredible third grader and his family have been on the field and TJ…


Added by Will Nolan on September 16, 2013 at 1:04pm — No Comments

Demystifying the FDA Meeting, September 10, 2013

For many years, patient representatives have been invited to participate in meetings around drug development as the patient voice is extremely important if reviews are to understand how patients weigh benefit and risk within the context of their disease. I was honored to present earlier this week at meetings for NINDS and the FDA. Below are some of the notes I took and some of the information I heard that I wanted to share with you all.


How does it work?  FDA issues guidance…


Added by Pat Furlong on September 13, 2013 at 2:21pm — No Comments

PPMD Leadership on Patient-Focused Drug Development Nets Invitation to Present at Two Influential Meetings

Parent Project Muscular Dystrophy (PPMD) has been invited to present at two upcoming stakeholder meetings.

The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of PPMD's groundbreaking work to advance the patient and parent perspective in the drug development and review…


Added by PPMD on September 10, 2013 at 9:44am — No Comments

Spiritual Journey to the Arches

Guest post by Jill Anne Castle, mom to Oliver, 11, and Anthony, 13 and living with Duchenne. Jill is an Educational Consultant and PPMD FACES of Arizona Volunteer Coordinator.

Spiritual Journey to the Arches…


Added by PPMD on September 9, 2013 at 8:30am — 2 Comments

How I Wish

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …


Added by PPMD on September 6, 2013 at 12:46am — 2 Comments

2013 MDCC Meeting

The Muscular Dystrophy Coordinating Committee (MDCC) had its 10th meeting on August 26, 2013 at the National Institutes of Health offices in Bethesda, MD.  The members of the MDCC come from federal agencies involved in one or more muscular dystrophy programs as well as public representatives from several organizations representing one or more forms of muscular dystrophy.  There are representatives from…


Added by Brian Denger on September 4, 2013 at 3:00pm — No Comments

Hi all members of PPMD community

Hi everyone in PPMD community. I would like to wish you guys all the best. My name is Doan - Leader of Vietnamese DMD Club, I have lived with Duchene muscular dystrophy for 30 years. Vietnamese DMD community was born with a mission of improving and developing life quality of…


Added by Doan Nguyen Xuan on September 4, 2013 at 4:05am — No Comments

Back to School - Duchenne Resources

It’s that time of year again – the start of another school year. While we put our faith in schools to provide a proper, inclusive education for our children, faith is not all we have in our arsenal for ensuring this is the case for students living with Duchenne.


As parents, you are constant advocates for your children; this is especially true with…


Added by Ryan Fischer on September 3, 2013 at 10:45am — No Comments

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