Prosensa Announces 48-Week Data from a U.S. Phase II Placebo-Controlled Study of Drisapersen in 51 DMD Boys

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My son participated in the GSK 51 trial for the last 2 years. We have noticed some improvement but overall he did not improve so much. We did see some strength gaining but lots of side effects.. Real and very worrying side effects. It is very sad that the drug is not showing the premise and promise so many of us put on it. I feel sorry the company is not being very honest with the public about the severity of the side effects. I personally not so sure if it was worth risking my son life. After all the drug has also the potential to shorten their life with these hope-full injections. He suffers from scars that can become a real problem if not treated and he had kidneys problems. We are not sure what they were and are. Well that is what we know for now. Who knows what others changes in the body genes and mechanisms for the long run can occur? We all want our son to be saved from the death verdict duchenne impose on us. As a clinical psychologist  it is intolerable for me to be normal while watching my lovely young boys dying day by day from dmd. I try to find meaning in dmd and in fighting it as an Israeli. We all know death is there just around the corner and for most boys it is very soon. This is scary.. So I tend to hold to these drugs. At times I feel it is wiser not to risk my son anymore with new complicated drugs, who's developments involved with politics and big money and can carry the potential to shorten my boys already shorten lifespan. As a religious Jew I cannot  refrain from listening to the heavenly voice that tells me day by day aren't you Mr. Levy, infected with the old curse of those people who built  the Tower of Babel trying to reach the unreachable? Where are the boundaries? Well I say to myself, maybe the issue is that I write these words from the Holy land to the PPMD in The Dreamland? After all who can watch her or his son die and do nothing? you are talking about 300K 400K whooo.. wait it is not woking yet man! don't rush you can also lose something, been there done that.

replys by mail: psy.levy@gmail.com

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Dori Levy

Dori,
Is it possible your son got the placebo instead of the drug? I heard the injection site reaction can happen even in the case of placebo.

Thanks,
Prakash
No no he got the drug. And that is why he had all these side effects.

The Sarepta drug is the better drug, the side effects much less but the FDA is treating them equally.  If they were both approved I would choose Sarepta.    

My son got Drisapersen for 2 years and did wonderfully on it with minor side effects, thankfully. He is 13.75 yrs old, and is having trouble getting in and out of the car and on the stairs. He had been stable for at least 1.5 years since starting in the trial. Really too bad to let him waste away now.

Andrea, have you looked into the blood checks personally? Or did you rely on what they told you? There were some serious side effects in the private tests we have been doing on my boy. Why did they stop a 400-600 M$ trial if it was working? (We all know it did work because we noticed the stability) It is not a minor decision as you know. I assume it was too dangerous to continue and they don't really tell us all they know. Maybe I am just paranoid.. Maybe. May G-d bless all our kids with healthy and long life.

Jason, I was told by one of the Dr that the Sarepta drug is not strong enough. One need to be careful and not believe anything you read over the web, most is PR deviated.

 

We think clinical trials are our hope, but when we are there it can also carry the potential to be the end, so take a real real care!

 

My sons regular test results were the  same as usual, except for urinary micro albumin, which always returned to normal when they would not inject that week. Some boys on placebo also had this happen, curiously. 

I have also thought that the boys on drisapersen who responded seemed to have a "stronger" benefit (videos of them riding bikes, etc) than the ones I have seen on eteplirsen, but that is just my personal observation. Not denying there have been some with bad side effects. But also aware that sometimes, to be on the safe side, researchers will call something an adverse event though they have no real proof that it wouldn't have happened without the drug. For instance, like the boys getting placebo had urinary micro albumin too. Also hard to say when the mix of placebo/delayed treatment is thrown in the mix, and the wider age range.

I've heard of 2 serious AE that required the child to withdraw from the study with GSK. What were the serious effects revealed by your private testing Dori? Was the doctor who told you eteplirsen was not strong enough part of the trial or how were they "in the know"? Just curious, as I heard the same thing, second hand, from one of the eteplirsen study physicians.

Hope they can get to the bottom of the phase III problems or Sarepta can get AA without further placebo trials.

If only every drug was totally safe and totally effective...but right now I will take what we can get. After all, a lot of us give steroids which have their own set of serious side effects with long-term use.

Go Race to Yes! 

A.


 
 Dori said:

Jason, I was told by one of the Dr that the Sarepta drug is not strong enough. One need to be careful and not believe anything you read over the web, most is PR deviated.

 

We think clinical trials are our hope, but when we are there it can also carry the potential to be the end, so take a real real care!

 

I am wondering if it is so good why did they stop it...

So what were the lab results that concerned you, that you had done privately for your son Dori? 

How old is he and how is he doing now?

At some point there were some changes in the structure of some red blood cells or they have susspected a change.. as far as I remember.. my concern was about a possible cancerous process. Second test did not show it thanks gd. I believe they stopped the trial because they were afraid of possible cancerous processes.. I believe they had some initial evidence foe this and twas too risky for them to continue.. Otherwise I can't buy the claim there were not a significant improvment, we all report a change. My 2 sons are 9 and 7 both have dmd same mu

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