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Permalink Reply by Tuyet Lan Thi Tran on September 25, 2017 at 4:44am

Dear Sulthana Shaik

If have any chance to save your kids , go ahead. I am always wish all the best to your son

My son also need exon 45 & 51 skipping but I am in Viet Nam , dont have chance for any trial and I dont know who to contact to get the trial.

Brgds

Lan

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Permalink Reply by Bruce Ward on September 25, 2017 at 10:18am

What would you have to give up to be in the trial?

Is your son on steroids or supplements that are working well?

If it is one of the Sarpeta trials have a look at the prescribing info for their Exon 51 product http://exondys51hcp.com/sites/default/files/EXONDYS%2051%E2%84%A2%2...

I read this recently and was shocked that Exondys 51 only increased dystrophin by ~1%. Perhaps that is a helpful amount but DMD patients are thought to have 0% to 5% normal dystrophin while Becker patients are thought to have 20% or more. My guess is that Exondys 51 would have minimal, perhaps weeks or a small number of months, effect on ambulation.

I hope I am wrong but I would not trade clinically documented supplements for Exondys 51. I hope and pray that their future products will produce better results.

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Permalink Reply by Kirubha Balakumar on September 25, 2017 at 6:28pm

We were not in trials. We started exon 51 2 months back. We do see a lot of difference since we started on exon 51. At 4 years old he had trouble climbing even a toy slide. But now he is climbing the slide in park in reverse. No falling anymore and is on his feet all day..

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Permalink Reply by sulthana shaik on September 25, 2017 at 6:48pm

Hi ,

How old was your son when you started on exon skipping?

How long did it take for the biopsy to heal?

Did he also start on the steroids recently?

Please reply.

Kirubha Balakumar said:

We were not in trials. We started exon 51 2 months back. We do see a lot of difference since we started on exon 51. At 4 years old he had trouble climbing even a toy slide. But now he is climbing the slide in park in reverse. No falling anymore and is on his feet all day..

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Permalink Reply by sulthana shaik on September 25, 2017 at 6:56pm

Thank you for your reply, It's a very difficult decision to make, not sure about the side effects,the biopsy etc

Don't want to give my son more pain. 
Bruce Ward said:

What would you have to give up to be in the trial?

Is your son on steroids or supplements that are working well?

If it is one of the Sarpeta trials have a look at the prescribing info for their Exon 51 product http://exondys51hcp.com/sites/default/files/EXONDYS%2051%E2%84%A2%2...

I read this recently and was shocked that Exondys 51 only increased dystrophin by ~1%. Perhaps that is a helpful amount but DMD patients are thought to have 0% to 5% normal dystrophin while Becker patients are thought to have 20% or more. My guess is that Exondys 51 would have minimal, perhaps weeks or a small number of months, effect on ambulation.

I hope I am wrong but I would not trade clinically documented supplements for Exondys 51. I hope and pray that their future products will produce better results.

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Permalink Reply by Kirubha Balakumar on September 25, 2017 at 8:29pm

My son is 4.9 years old and we started exon 51 two months back. We didn't do muscle biopsy only blood test. He started steroids 4 months back.

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Permalink Reply by sulthana shaik on September 25, 2017 at 9:48pm

Have you noticed any side effects? Do you need to do kidney tests while on exon 51?

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Permalink Reply by Kirubha Balakumar on September 25, 2017 at 10:56pm

Only dehydration. But we're managing with lots of Water. Haven't done any kidney tests yet.

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