Zane Braun of Whitechurch suffers from Morquio A Syndrome, a rare genetic disorder that will shorten his life. The drug will potentially decrease the amount of sugars in their bodies and hopefully will slow down the progression of the disease. Luke and Zane Braun are seen enjoying some Christmas cheer.
The last 121 weeks for Luke (16) and Zane (14) Braun of Whitechurch have been positive in the eyes of their doctors said their mother Debbie Braun has also seen improvement in their energy levels. The two teenage brothers are going through a clinical drug trial to help slow the advancement of Morquio A Syndrome. It’s a rare genetic disorder that will shorten their lives.
Luke and Zane are missing an enzyme that breaks down complex sugars causing dwarfism, respiratory problems and extreme exhaustion. Approximately 3,000 people are living with Morquio A Syndrome around the world.
The boys are part of a few who are testing the drug, Vimizim, in Canada, which was recently sent to the US Food and Drug Administration (FDA) in the US for approval as well as it being sent for approval to Health Canada. Debbie said she has seen a marked improvement in both her sons’ energy levels. She said they are able to go through a whole day without having to rest.
“All of the different results the doctors have come up with for the last 121 weeks have been coming up positive especially from their lung function tests and with their endurance levels. Everything is all positive so they have decided to send all of the paperwork off to get approval from the FDA and Health Canada,” said Braun. “This drug will slow the progression of the syndrome down and make their lives more manageable.”
Metabolic geneticist Dr. Julian Raiman said the drug will decrease the amount of sugars in their bodies and will potentially slow down the progression of the disease. Raiman said he has seen a significant improvement in their testing. One of the tests he said tests their endurance by climbing stairs. He said they are able to do that at an increased efficiency.
Debbie said her boys have been able to be more like regular teenagers since they have been on the drug. They are able to stay up late like many teenagers do and she said the added energy has given them the ability to be more attentive at school as well. Both of the boys attend FE Madill School in Wingham. Zane is in Grade 9 and Luke is in Grade 11.
“Before they were on the drug the boys were doing a lot of napping during the day because they were exhausted. They would often fall asleep on the bus on the way home from school as well as after dinner a lot of the time. I’ve certainly noticed since they have been on the drug that they are much more energetic and can stay awake for the whole day,” said Debbie. “Luke has done really well in school especially with the amount of time missed being in Toronto doing the drug trial. Zane has also been a lot of more attentive at school and his teachers have said and he is learning a lot better because he’s not so exhausted when he is in class.”
Debbie said the drug trial has given her and her family hope for the Luke and Zane’s future. The treatment isn’t a cure, but Debbie hopes it will give her sons time to live their lives as independent well-adjusted people.
“When the boys were first diagnosed I was told it was a progressive degenerative disease and that at some point the boys would pass away and wouldn’t live long,” said Debbie. “The treatment has given us more hope and it makes the quality of their lives better, which is important to me. I just want them to be as independent as possible and it makes me so proud to see where they are in their lives right now.”
Debbie said one of the big reasons Luke and Zane went on the drug trials was to improve their condition, but to also provide help for the other people who have Morquio A Syndrome. It’s another point of pride for their mother. She said the boys are very selfless people who never seem to get down due to their situation.
“One of the reasons we started this drug trial wasn’t just for the boys and I think that was one of Luke and Zane’s goal started. They do want to live longer, but they also want to give all of the friends they have met with Morquio Syndrome to have access to the drug as well,” said Debbie. “Right now Luke and Zane are on the drug and they have access to it, so getting approval for it is extremely important to us. Luke and Zane are very kind hearted and they want to help everyone they can through this drug trial and they make me so proud everyday because of things like that.”
By Garit Reid, Grey-Bruce This Week
SOURCE: http://www.lucknowsentinel.com/2013/12/17/drug-trial-for-whitechurch-family-a-sign-of-new-hope