September 9, 2006 mcfadyena

Where We Are at Today

Where to begin… Pretty much has soon as we found out Trey had Hunter’s, we found out that there was no treatment available yet, but that there probably would be soon in the U.S. The treatment is an Enzyme Replacement Therapy (ERT) called Elaprase that treats the physical symptoms of Hunter’s. It is an Orphan Drug, and since Canada does not yet have an Orphan Drug policy, we found out that it will be hard to get it in Canada.

Elaprase was approved by the FDA in the US on July 24, 2006, which is awesome. This means that we can apply through Health Canada for Special Access to use the ERT in Canada. Apparently this part of the process is not extremely difficult, the difficult part is finding someone to fund  the ERT. The cost of Elaprase depends on the weight of the person using it, but the cost will definately be in the hundreds of thousands per year.
Our doctor, Dr. Sylvia Stockler, who works out of BC Children’s Hospital, has already applied for Special Access for Trey and has also applied to both the drug company, Shire Pharmaceuticals, and BC Children’s Hospital for funding. We should hear back about Special Access in the next few weeks and we will hopefully hear back about funding within a month.
We have also been in contact with our Minister of Health, the Honorable George Abbott, and his Assistant Deputy Minister of Health, Bob Nakagawa, to introduce them toTrey. In the long term it is our government whose support we need for everything about Trey’s MPS.
We will need their help with funding the drug for Trey, raising awareness about MPS, and from a larger perspective, we need their help in creating an Orphan Drug policy so that in the future, people with rare diseases will not have to jump through the hoops that we are having to jump through in order to get help and treatment.


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