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Sarepta Exon Skipping 45 amenable - limited or no ambulation dose titration study is recruiting

Dear Friends,

Sarepta is running a trial at 3 sites recruiting a total of 12 patients for a dose escalation study of 12 weeks followed by open label extension of their exon skipping 45 drug SRP-4045 for boys who are non-ambulatory or have limited ambulation (age 7 to 21). The clinical gov link for the study is https://clinicaltrials.gov/ct2/show/NCT02530905?term=sarepta+duchen...

The site in Chicago is Lurie Children's that has started recruiting. Contact would be Kiera Jackson (KiJackson@luriechildrens.org)

The other 2 sites per clinicaltrials.gov are David Geffen School of Medicine at UCLA and Kennedy Krieger Institute at Maryland.

If you or your child seems eligible, you should contact the nearest site immediately.

To directly reach Sarepta, contact Kara Boniface (KBoniface@sarepta.com)

Best wishes and stay blessed

Alpa

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Permalink Reply by Rahul Deshpande on November 16, 2015 at 11:59am

Don't know when they will start the study for ambulatory boys. This drug will be used for the 2nd highest percentage of DMD population but somehow not seeing any trial from Prosensa or Sarepta for ambulatory boys.

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Permalink Reply by Alpa Khushalani on November 17, 2015 at 11:40pm

Hello Rahul,

Sarepta plans to start trial for ambulatory boys hopefully early 2nd quarter of 2016 per my discussion with them. My son Krishna is 8 and ambulatory so I'm also eagerly waiting for the trail to start.

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Permalink Reply by Rahul Deshpande on November 18, 2015 at 8:17am

Hello Alpa,

You are right the trial will hopefully start in 2nd quarter, yesterday I got a call from Sarepta and they said that the trial will start sometime in 2016. My son is 10 and ambulatory having 44 deletion hence eagerly waiting for 45 skipping trial. We should also keep track on Prosensa also as they have started a 45 skipping trial in Europe.