Wait four months to join clinic trial or start with prednisone now

My son Max was diagnosed with DMD less 2 weeks ago. It was hell to swallow this while keep tracking all relevant information for his treatment. 

We now face a dilemma and would like to hear your suggestions based on your experiences. Max is 4 and since he has not taken any medication he is eligible for two clinic trials in UC Davis where the medicine in test are supposed to have less side effects - called Vamorolone and Catabasis if anyone came across it. Both trials are for about 1 year. 

The issue is Max will be out of country this summer for about 2 months, so if we want to register him for the clinic trails he wouldn't be able to start until August. That means waiting for 4 months without taking prednisone as it will DISQUALIFY him from the trials. 

Shall I wait for it or start with prednisone right away? He is young and so far no obvious difficulty in mobility, but easy to fall and cannot run much nor jump. How important is it to start meditation as soon as possible, especially knowing there are side effects? Does it worth delaying his meditation for four months for the POTENTIAL benefits of clinic trials? Please feel free to share your thoughts and experience. As a heart-broken father, any words from this community will help. 

Thanks a lot! 

Tao

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Very sorry to hear this.  I can still vividly remember the 'punch to the gut' when we heard the news about our son almost 10 years ago.  However, you've found a great community in PPMD.

PPMD recently published a 10-year study on Duchenne and I recall a chart showing the significant benefits of steroids.  Deflazacort had even greater benefits over prednisone.  (We originally purchased deflazacort from the UK for about $50/mo, but PTC launched the identical product in the US, increasing the cost to over $7000/mo!  Describing this move as unethical and greedy doesn't begin to capture the anger.)

You'll have to make a personal decision regarding clinical trials.  Our son hasn't been eligible for many of them and the uncertainty of the benefits of the drug, the 'risk' of getting a placebo and missing out on steroids has always been concern for us.  But please don't let our decision influence yours.  Every situation is different.

One thing we've learned is to treasure each and every day with your son.  Yes, it is hard (very hard some days) but life can still be great.  Also, find a medical care team that specializes in Duchenne.  We visit Cincinnati every year but there are many clinics that PPMD has certified.  There are very unique requirements for Duchenne that a general care facility will not know.

Thank you Keith and Jill, sorry to hear that your lovely son Chase has already been on this difficult path for 10 years! I hope he is doing well, and I know he is. 

Your words are very comforting. I can't really think about future since I got the news, but knowing that you have dealt with for more than 10 years and can still remain positive, hopeful and passionate is a great encouragement to me. Thank you! 

I spoke with my doctor again, and will follow her suggestion on this to go on steroids first. The concerns you mentioned were also raised by her as well. I also felt quite luck there are great provider, supportive facility and community here in the bay area for DMD patient and family. I know it is going to be hard, very hard, but I will keep reminding myself to remain hopeful and treasure every single day with Max.

Thank you! 

Keith & Jill VE said:

Very sorry to hear this.  I can still vividly remember the 'punch to the gut' when we heard the news about our son almost 10 years ago.  However, you've found a great community in PPMD.

PPMD recently published a 10-year study on Duchenne and I recall a chart showing the significant benefits of steroids.  Deflazacort had even greater benefits over prednisone.  (We originally purchased deflazacort from the UK for about $50/mo, but PTC launched the identical product in the US, increasing the cost to over $7000/mo!  Describing this move as unethical and greedy doesn't begin to capture the anger.)

You'll have to make a personal decision regarding clinical trials.  Our son hasn't been eligible for many of them and the uncertainty of the benefits of the drug, the 'risk' of getting a placebo and missing out on steroids has always been concern for us.  But please don't let our decision influence yours.  Every situation is different.

One thing we've learned is to treasure each and every day with your son.  Yes, it is hard (very hard some days) but life can still be great.  Also, find a medical care team that specializes in Duchenne.  We visit Cincinnati every year but there are many clinics that PPMD has certified.  There are very unique requirements for Duchenne that a general care facility will not know.

We're sorry to hear this too. Keith and Jill, your reply was spot on. Everything you wrote was what we've experienced. Tao, the first couple years are the worst. But yes it does get better. Our 14 year old son loves life. He's had an incredible impact on so many people that never would've taken place if Duchenne weren't part of the picture. Be assured your son is making a difference in our world too. Hold on to that.

Re: the clinical trial decision, we suggest keeping up the dialogue with your neuromuscular doctor (if you haven't already done so). In our humble opinion, since it's such a new diagnosis, starting steroids is probably the best bet until things have settled down.With that said, assembling a neuromuscular team is crucial. It's worth every penny to travel to a certified Duchenne Care Center (we travel from Arizona to Cincinnati Children's Hospital every year - though CCH lost their amazing lead doc, Brenda Wong, to a new Duchenne Center in Massachusetts, the team at Cincinnati is still pretty incredible). Whatever team you choose, they can help guide you in clinical trial decisions in the future.

Godspeed as you and your family navigate this new road.

Shawn & Andrea

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