We are planning to participate in exon 45 skipping trail, for our 12 year old son.
Is any one already enrolled in the exon skipping trail for either 51/45/53, Can you please share your experience. Struggling to decide if it will be the best decision for our son.
Are there huge risks?
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.
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?
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..
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.