Featured Blog Posts – September 2013 Archive (5)

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

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

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

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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