I’m sitting at the National MPS Society conference and learning more about the next step in the testing of Idursulfase IT – the Phase III trial. I know families are anxiously awaiting information so I wanted to push this out there as soon as possible. If you are looking for your child to enter the trial, my advice would be to contact the expert MPS II physician at the location nearest you. I understand that the cognitive testing requires the child/parent to be proficient in English or Spanish as well. The non-U.S. sites are expected to begin first. This is the other information I have gained.
AIM-IT: Administration of Idursulfase in MPS II – Intrathecal
I hope this information is helpful and my heart is with those seeking to enter the next phase of the trial and find stabilization and a longer life for their children. Clinical trials are not for the faint of heart and I’d be happy to discuss some of the logistical challenges we faced and how we made it all work. As always, I try to be an open book.