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

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Same mutation.
My older one is not doing well he walk with difficulties. He was stable for the last 2 years and now started to fall more.
It was my concern based on hematology findings that did not appear in a second blood test .
I will try to find the specific test and share it...

I work in a pediatric lab, and there can be little red flags that upon further investigation are nothing, caused by a fussy ananyzer, poor blood draw, etc. One thing, a cancerous process would not usually alter red cell structure. I had heard of some coagulation problems, but don't see them listed in the latest phase II and III results I have seen lately, so perhaps it was in much older data? So much to read and remember...

Peace to you you and your family Dori.

The baseline 6-Minute Walk Test went from >390 meters in phase 2 to <350 meters in phase 3 this could have lead to the failed phase 3. I would like to know, why the inclusion criteria was changed so drastically from phase 2 to phase 3?    Did the FDA or GSK make this change?  It's like the shot themselves in the foot. Now it looks like the FDA is pushing Sarepta in the same direction. I hope Sarepata can resist such a change in light of the foolish notion by the FDA that DMD patients with a "baseline 6-Minute Walk Test (6MWT) ≥350 meters predicts continued general stability"


Well my son had at some point evidence of blood coagulation and they did re checked it and it wasn't there thanks g-d but it did appear in some boys tests. Just to remind you that blood coagulation are found to be connected to cancerous processes and let me quote you some things about Coagulation taken from the American Society of Clinical Oncology (ASCO):


"Coagulation is the term used to describe the clotting of the blood…."


"A blood clot (also called a thromboembolism) is a serious side effect of cancer and cancer treatment. A blood clot that develops in a vein (a blood vessel that carries blood to the heart) is called a venous thromboembolism (VTE). It may develop in any vein but happens most often in the legs, thighs, or pelvis (lower abdomen). This is called deep vein thrombosis (DVT). A pulmonary embolism (PE) is a blood clot that has traveled to the lung from another place in the body. A PE can be life-threatening.

Blood clots are common in patients with cancer. Many patients are not aware of the risks, symptoms, and signs of a blood clot. The risks for a blood clot are listed below, followed by the signs and symptoms. If you have questions and concerns about the risks and symptoms listed below, it’s important to talk with your doctor.

Risk factors for blood clots

  • Cancer, especially cancers of the lung, kidney, brain, digestive system, female reproductive system (such as uterine cancer), and blood (such as leukemia and lymphoma)
  • Metastatic cancer (cancer that has spread to other parts of the body from where it started)
  • Cancer treatment, including surgery lasting longer than one hour, chemotherapy, radiation therapy, and hormone therapy. Antiangiogenic drugs (drugs that block the formation of new blood vessels), such as thalidomide (Synovir, Thalomid) and lenalidomide (Revlimid), may increase the risk of blood clots.
  • Treatment with erythropoiesis stimulating agents (drugs that help the body make more red blood cells), such as epoetin (Epogen, Eprex, Procrit) and darbepoetin (Aranesp)
  • Being in the hospital, because most patients in the hospital can't be physically active and need to stay in bed or are unable to get out of bed  

Again, if we all agree that our boys did show impressive improvements so how come a corporation like GSK drop the golden egg so quickly? Do you really believe they are going to go to the public and tell us that there was a risk of cancer? Of course not, because there wasn't any real cancer yet, but anyone neutral, cold blooded business man, (not like us, that carry in our mind the death verdict imposed on our lovely children) any corporate business man from that corporation would say a big no no to these findings. Now I assume they are not going to tell the public this is the case. No no. They are going to reason it with statistical reasoning explaining why they have decided to drop 400m or 600m that're invested in the 51 exon skipping...  Just like that..

Just to show you the integrity STYLE of the GSK Corporation:

14 patients whose drug for treating hypothyroidism was reformulated without their knowledge file class-action suit.

The lawsuit is aimed against Perrigo and GlaxoSmithKline Israel, and two health funds – Clalit and Maccabi Health Services. The lawyer has asked for NIS 4.5 billion in damages for his clients.



GlaxoSmithKline to pay $3B in health care fraud suit


Glaxo to Pay $750 Million for Sale of Bad Products

so I guess the question for me remain what do they don't tell us? If the oligos are so good why stopping?





 And from the Prosensa webinar I learn that the little sister is learning VERY QUICKLY step by step to walk the talk of the big sis, VERY VERY BAD! indeed

And please don't get me wrong I am willing to do all I can to save my kids life

People get clots from long plane flights too. 

I had issues with the way the blood draws were being performed for the coagulation testing by some trial nurses (leaving the tourniquet on for way too long causing hemostatis and not taking the tubes in the correct order). I brought it up and the one nurse who was really bad at it did not draw blood from my son again, sure she did on the other participants.

I don't think they would knowingly leave themselves vulnerable to future law suits by purposely hiding cancer risk. None of the boys treated long term (4+ years) have gotten cancer, and let's face it, some could without exon skipping drugs, DMD patients are not immune to contracting other diseases. Did any of the animals studied get cancer? It's clearly explained in the consent form that the main problem could be kidney damage. Don't think they would warn of that but not cancer, both are serious if it happens.

You say GSK dropped the trial "just like that", but their 5 year contract was over, no? (2009-2014) Perhaps like Sarepta, they thought they could get accelerated approval before now, but unlike Sarepta, they didn't say it to the community. This is just my opinion. Prosensa (with GSK anyway) just seems to do "business" differently than Sarepta.

Sorry can't buy this, you don't just leave behind couple of hundreds millions just because contract is over.. no no

So we disagree, that is ok, I guess time will tell us more meanwhile I pray for good health to all our boys


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