http://www.prosensa.eu/news/TREAT-NMD_Prosensa.pdf

Prosensa and TREAT-NMD enter into strategic collaboration
for PRO-051 clinical trial planning

Leiden, August 31, 2009 – Prosensa, the Dutch based biopharmaceutical
company focusing on RNA modulating therapeutics, announces the successful
completion of a feasibility enquiry using the TREAT-NMD Global Database for
DMD and the TREAT-NMD Care and Trial Sites Registry for the planning of the
phase II/III study for its lead compound PRO-051.
Prosensa recently completed a phase I/II clinical trial for PRO-051, its lead compound
for the treatment of Duchenne Muscular Dystrophy (DMD), and the company
anticipates starting a phase II/III clinical study early next year. PRO-051 is directed to a
specific mutation in the dystrophin gene that occurs in approximately 13% of the DMD
patient population. In order to set up the pivotal study, patients with a genetic mutation
amenable to exon skipping by PRO-051 need to be selected.
TREAT-NMD has developed a global database that contains precise genetic and
clinical information from patients with DMD, including age, ambulation status and
medication use. Using the TREAT-NMD Global Database for DMD, Prosensa has
identified around 300 patients from 21 countries who meet the inclusion criteria for the
upcoming trial. The TREAT-NMD database holds up-to-date information about all these
patients that will allow them to be contacted for trial recruitment purposes through the
national registries. These patients were matched to 50 potential trial sites and selected
patients and sites will be contacted for participation in the upcoming clinical trial.
“Patient recruitment for clinical studies is a very expensive and time-consuming
process, in particular for rare diseases such as DMD” comments Dr. Giles Campion,
CMO and VP R&D of Prosensa. “Therefore, this successful collaboration with TREATNMD
is extremely valuable for us as it facilitates the acceleration of the recruitment
process and hopefully allows us to bring this treatment faster to patients.”
“We are delighted that our global patient database is providing such useful results for
pharmaceutical companies like Prosensa,” said Professor Hanns Lochmüller, leader of
the TREAT-NMD patient registries initiative. “Our approach is unique because the data
we are giving companies isn’t just generic statistical information. It is up-to-date
information about real patients who can be recruited into clinical trials through the
registry or contacted when a treatment is available. At the same time, it’s safe for
patients, since we don’t give out identifiable information to companies but act as a
trusted intermediary and all our registries have full ethical approval and comply with
data protection laws.”
Prosensa and TREAT-NMD will both be attending and presenting at the World Muscle
Society Conference in Geneva from September 9th till 12th.

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All,

I hate that there is more than one registry. How many are there and how do you learn about them?

I believe MDA has one, Utah and there is Duchenne Connect. Why can't there be one central registry? Aren't we all in this together and trying to make things simpler for our families? Life is complex enough. Sorry for the venting. I HATE duplication and believe in standards of care for our families.

Now, seriously how do we learn about all the different registries?

Thanks all!

Julie
Thanks for the responses, I get frustrated that I am busy being a mom, i have registered the boys with duchenne connect, I was told they all share info. I also contacted dr wong who sees the boys annually. Her nurse wrote back that they will "keep us in mind" when they hear about prosensa trials. Should I contact prosensa too? I am so beaten down, 10 years of calling, emailing , begging people to help my sons out, I am seriously tired of being a pushy nagging mom! LOL thanks for the support, nobody else gets it like you all do.
It is my understanding as well that each registry shares information. But I am also registered with connections all over the world including Germany, Italy and the UK. I wish there were just one as well, would make life so much easier. We, as parents, will be the best advocates for our kids with MD - not a registry. If we leave things in the hands of others, things can slip through the cracks. Chances are that US boys will be taken as a last resort for trials in Europe because they have their own set of boys eligible for their trials. But for the US trials - especially those concerning exon 51 skipping - they will collect kids from US registries - not European ones. Anyone can look to the inclusion/exclusion criteria for past trials. For US exon 51 skipping, they will look for children with confirmed DMD diagnosis through biopsy. Be fully prepared. Hang in there. Everyone!


jenn said:
Thanks for the responses, I get frustrated that I am busy being a mom, i have registered the boys with duchenne connect, I was told they all share info. I also contacted dr wong who sees the boys annually. Her nurse wrote back that they will "keep us in mind" when they hear about prosensa trials. Should I contact prosensa too? I am so beaten down, 10 years of calling, emailing , begging people to help my sons out, I am seriously tired of being a pushy nagging mom! LOL thanks for the support, nobody else gets it like you all do.

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