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

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

The other 2 sites per 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 (

Best wishes and stay blessed


Views: 707

Reply to This

Replies to This Discussion

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.


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. 

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.

Any news on the 45 skipping trial. Did they start screening children for the trial.

I am curious as well
Please check with nationwide.. They might recruit for 45 within 4-5 months.

Reply to Discussion


Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service