Spoke with Dr. Muenzer Regarding IT Trial Set-back

I spoke with Dr. Muenzer today regarding the IT Idursufase trial not meeting it’s endpoints. If you have any questions regarding the future of the trial, please feel free to reach out to myself or Dr. Muenzer. If you don’t have Dr. Muenzer’s contact information, I can provide you with it (he gave me permission). For any family in the trial or for concerned extended family or community members, he would like to provide as much information as he can in order to relieve anxiety, so please don’t hesitate in reaching out. The trial did not meet its endpoints, but this does NOT mean the drug does not work and it does NOT mean the trial is shut down. Dr. Muenzer knows the drug works. He sees our kids and he knows where they would be without the drug.

People have pointed out to me in the past day that gene therapy is coming up if this trial does get shut down. One: this trial is not getting shut down unless it’s over my jailed or dead body. Two: Gene therapy is hopeful and likely, but I will not rest my child’s life on hope and possibility. Nobody knows this better than families who have kids with MPS II Trey’s age and older. When Trey was diagnosed at the age of two in 2006, IT trial research was already going on. I spoke with Dr. Muenzer about it every few months. Every spring we were told human clinical trials would begin in the fall. Every fall we were told it would begin in the winter. Every winter we were told it would begin in the spring. For three years. During that time, most of Trey’s peers fell outside of the IT trial criteria and therefore did not qualify.

So yes, gene therapy will hopefully come to fruition, but I will not let a drug that is holding my child stable and saving his life, go, with the hope for gene therapy. I don’t know gene therapy will come, and if it does come, I don’t know if Trey will qualify for the trial, and I don’t know if it will come in time to save Trey.

Trey has IT therapy. I am not giving that up until or unless there is another treatment that is available and works.

With love,

Deb

IT Idursulfase Trial Faces Setback

Please read:

https://endpts.com/shire-hit-by-a-setback-as-hunter-syndrome-drug-fails-late-stage-study/

https://www.shire.com/en/newsroom/2017/december/wvdwq3

I’m not going to say a lot right now as I’m still processing this news and I’m not sure how much I can/should/want to be posting on the big wide web. What I do know without a doubt is that this drug works and I will go to jail before I see this drug get taken away. Melissa Hogan has issued a Clinical Trial Alert that contains more information, please see below.

ClinicalTrialAlert-SHP609

If you have questions, don’t hesitate to reach out.

Love Deb