Elaprase has been approved by Health Canada!! This is a huge step because it means that Elaprase is now accessible to all people in Canada affected by Hunter Syndrome. It was approved on June 14. The catch is that although it means that the drug is approved for use in Canada, it is up to the individual provinces to approve funding. This, as has been demonstrated with many rare disease drugs, is where the struggle (and frustration and despair and and and) comes in and this is why Canada is so badly in need of an Orphan Drug Act. As soon as I have a final copy of the Press Release, I will post it up here. And speaking of Elaprase, on Monday, Trey completed his 20th infusion!! What an absolutely great feeling.
We have also received a few more results from Trey’s recent series of tests. His abdominal ultrasound (which was done in May) showed that his spleen (which was enlarged before he began ERT) is now within the limits of what is defined as normal for his age. His liver, which was 1.7 times the “normal” size before ERT, was described as bulky, which from my understanding means that it is still a bit big, but much smaller than it was, and almost within normal range. Trey’s doctors expect that it will continue to shrink. They also said that ultrasound is just a guide and not as accurate as an abdominal MRI, which will be done in December.
Trey’s cardiology results. A bit confusing. His electrocardiogram was normal. His echocardiogram report showed that he has trace Mitral Valve Regurgitation and as a result, his left ventricle is a bit larger than normal. I was told one year ago that Trey had Mild Mitral Valve Regurgitation (so trace MR is an improvement), however, the report from last year does not say anything about MR (I would have had no idea what MR was unless someone told me my kid has it, so I know the words were mentioned to me). The cardiologist who works with kids with MPS at BC Children’s is on holidays, so I’ll find out more in a few weeks. With what we are working with so far though, it seems as though Trey’s heart has not gotten any worse, and with a progressive disease, I am pretty thrilled with these results.
