2nd child getting sprt treatment shares improvement

http://www.kfvs12.com/story/19411932/clinical-trial-shows-promise-f... Video



Even with Duchenne Muscular Dystrophy, there's no stopping 12 year old Justin Trovillion. We met Justin last year just before he took part in a trail involving an experimental drug.

His mother Carrie Trovillion says she feels since the trial, Justin has a new energy for life.

"Now I'm pulling him inside at night trying to make him go to bed!," said Carrie. "He doesn't want to sit on the couch anymore!"

Now swim therapy in Cape Girardeau is one of Justin's favorite things.

"I usually swim ten laps across the pool and usually stand up on the side and sometimes we play ball," said Justin.

Carrie says that's a lot more than Justin used to do.

"He is able to do so much more," said Carrie. "That's what I see with my own eyes. What we do at home is a big part of it but with this new drug I feel like I see more energy. That's just me as a mom. That's what you want for your child."

Justin was one of 12 patients chosen nationwide for a clinical trial of the drug known now as Eteplirsen from Serapta.

"When he started taking the medicine we really started noticing more of an endurance and energy," said Carrie. "He feels good," said Carrie.

Children in the trial were either given a placebo, 30 mg's of the experimental drug, or 50 mg's. Carrie says Justin got the highest dosage of 50 mg, an answer to her prayers.

In the walk test, those kids walked further than the other children who didn't receive as much. Carrie feels that's just the beginning. She says they are waiting to find out if the drug will be approved.

"Now with the drug, you have more of a promise than just the hope," said Carrie. "We feel like this could give him a longer better life."

Right now Carrie says Justin's quality of life is better than ever. With new energy they recently participated in the 5K to support Mississippi Valley Therapeutic Horsemanship.

"It was just so fun," said Carrie. "We are addicted now."

Justin loves to ride Siri, a horse at MVTH. Exercise with Siri also helps Justin improve his muscle strength.

"He always says I just want to be more like the other kids," said Carrie. "You know we just want him to be happy."

"I can't wait to start horse therapy again," said Justin. "I like swimming, I like playing basketball and reading books sometimes. "

Carrie says thanks to the Muscular Dystrophy Association and all the money donated Justin gets to be just like any other 12 year old.

"The support is almost like counseling," said Carrie. "They get it. All the donations mean so much. As far as our family goes, we'd be lost without MDA."

Online: Cure Duchenne Blog


Apparently it is also being reported elsewhere that the trial will continue at least one more year and that they are adding local hospitals to provide treatments for some of the kids in the trial.



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That is SRPT not sprt.

The MDA has given Sarepta 0 dollars.

In all fairness it was the MDA which funded Dr. Wilton of Australia in the original research (yes he is the research hero in exon skipping) Plus the MDA did fund the travel costs for the current SRPT trial, plus they may have funded some of the costs associated with the lead investigator & Ohio hospital costs (not sure about that part).

However while MDA has not funded actual clinical  or SRPT development costs or trials, perhaps the opportunity/time has come for them to do so.

While not speaking for her, the mother may  be praising the MDA for all of the other assistance she has gotten up to this point.


I would hope that everything being consistant with the trial results that the MDA would join with all concerned to encourage the FDA to approve the SRPT approach  as a "drug class platform"  which could open the floodgates of funding and rapid development for all of the faulty exons. I believe that the FDA should realize that there would be no downside to such an approval.





One could argue without the long history of the MDA, Sarepta would likely have decided NOT to pursue Duchenne as the initial application for the platform. It's not as if they didn't have other options!

I haven't even watched the Telethon in YEARS

I will be glued to TV this weekend (and for once I don't mean football).



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