This is a good step. Last May, this finally went up. Hopefully, it will clear the way for the one so many of us are waiting for...

GLAXOSMITHKLINE announces a Phase I clinical trial for the investigational compound  GSK2402968 (formerly PRO051) in Non-ambulant DMD Subjects
(DEMAND I)

A new clinical trial for the investigational compound GSK2402968 (formerly PRO051) was posted to www.clinicaltrials.gov on 5/24/2010. This study, GSK
protocol # DMD114118, will assess the pharmacokinetics, safety and tolerability of GSK2402968 after a single administration, at escalating dose
levels, in non-ambulatory boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip. This study is not yet open for participant
recruitment. As the clinical development program advances, further clinical trials will be posted to www.clinicaltrials.gov and updated on the
DuchenneConnect website.


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we are keeping our fingers crossed that austin will get in, anyone have thoughts on whether participation in this trial will exclude participants from future phases in this trial (phase 2 and 3) ?
5 years after diagnosis I'm still struggling to correlate the information about the exon-to-be-skipped, and the out-of-frame deletion my son has.

Can anybody help? Boils down to this:

My son has out-of-frame deletion at exon 17. Does this make him a candidate for this GSK trial which skips exon 51? I've seen some information that suggests "yes" other that says "no."

Any feedback is welcome.
Unfortunately not. According to the trial serve predictor, your son will need skipping exon's 12-16 or 18 to restore the reading frame. You can check it out here.:


http://www.dmdregistry.org/reports/predictor/






David said:
5 years after diagnosis I'm still struggling to correlate the information about the exon-to-be-skipped, and the out-of-frame deletion my son has.

Can anybody help? Boils down to this:

My son has out-of-frame deletion at exon 17. Does this make him a candidate for this GSK trial which skips exon 51? I've seen some information that suggests "yes" other that says "no."

Any feedback is welcome.
Thank you Sharon, I had not seen this tool before. I didn't think he would qualify for this trial, but I could not make sense of all the numbers tossed about.

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