Final report on Idursulfase IT Trial ( final outcome to be reported on the Channel 7 program Australia)

21/06/2014 09:15

We are pleased to provide an update on the progress of the Intrathecal IT trial for MPS-II. The trial site selection has now progressed to the next stage and currently going through the “eligibility assessment” for running the trial. Shire is also consulting directly with the LSDP and has now provided all the information recently requested by them.

We are in regular contact with Shire and our local Federal MP who is communicating with the Federal Health Minister. We have been informed that the clause in the LSDP guidelines that doesn’t allow children currently receiving intraveneous ERT to participate in the Intrathecal IT trial is just a misunderstanding. It is now in the process of being confirmed in writing that this is not an issue. We will report on the final outcome of the trial with the Channel 7 program in July or in the near future. The program will report on the work that we have been doing from when we initially approached Shire in the United States 12 months ago. Also on our struggles to save our son who is making progress in some areas but losing vital skills in others.

The next focus for us post the intrathecal IT trial will be to find a cure for our son