Hope4Michaela Clinical Trial travel
Donation protected
Updated: May 2021
Short Version: Michaela’s time is running out. Many children lose their battle with Sanfilippo Syndrome in their early to mid-teens. Michaela continues to fight and we are so grateful that she is beating the odds. She will be 17 in August. The reality of this Boogie Man, that has haunted us for more than a dozen years, is closing in on us. We have a renewed hope and another chance to carry on this fight BUT WE NEED HELP.
Originally, Michaela was invited to participate in a clinical trial at Columbia University Medical Centre, Manhattan, New York that was scheduled to begin in May 2017. Due to money matters, corporate support of this trial was canceled. Lucky for us, TEAM SANFILIPPO stepped in to revive our hope and to make this clinical trial a reality.
In spring 2021, they began treating patients in New York with favourable results. We are looking forward to Michaela starting in 2021 at an upper Michigan site. This treatment has the potential to greatly improve Michaela's quality of life or perhaps even reverse some of the damage caused by Sanfilippo Syndrome. Most medical costs are covered but travel expenses are not. The trial itself has been extended to a 12 month study of WEEKLY infusions. All of the travel expenses are our responsibility.
I find myself overwhelmed by mixed emotions. I’m daring to HOPE for a better life for Michaela. I'm EXHAUSTED by the 13 long years that we've already fought this war knowing full well that the toughest battles remain ahead of us. I'm GRATEFUL for all the love and support throughout these years from our community. I am HUMBLED to be here again, begging for your help and FINANCIAL SUPPORT.
Our Story can be found HERE    .
The ORIGINAL DETAILS: On March 1, 2017, the house phone rang at a very early hour which startled me out of a deep sleep and immediately, that driving force kicked in. You know the one that bolsters you into fight or flight mode because instinct tells you that this call is going to change your life in some important way. Good or bad, it will be a call to action. Thankfully, this was a good news call, one that we had waited more than 8 years to get.
To completely understand its meaning, you have to go back in time to a very different phone call that we received in September of 2008, shortly after Michaela's fourth birthday. It was a call that takes your breath away and leaves you stunned, completely dumbfounded as you try to comprehend the message, frowning, shaking your head, unbelievable. There must be some mistake. Three years of questioning doctor after doctor and a summer of submitting this precious baby girl to poking, prodding and every test they could think of came to an end on that day, we had been searching for an answer and here it was. Michaela has mucopolysaccharidoses (MPS) type IIIB also known as Sanfilippo Syndrome. It is a very rare genetic disorder in which one tiny enzyme is mutated and therefore cannot break down a long chain sugar that is naturally manufactured in the body. These molecules then build up and cause progressive damage. With MPS III B, it begins in the central nervous system. Typically, by age 6, effected children lose their language, both the ability to speak and comprehend. By age 10, they are often wheel chair bound. Piece by piece, their abilities fade away leading to dependence on g-tubes and breathing machines until they leave this world sometime in their mid-teens to early twenties.
This was the bomb that devastated our world. How do you process the fact that this is the future of your beautiful child? After the initial shock wears off, fight or flight kicks in and questions take form. What do we do about this? How do we fight back? How do we save our baby? Who will help us? We were told. "There's nothing you can do. There's no cure. There aren't even any treatments. Just take her home and enjoy her while you can. She's not bad right now but it's coming."
So what would you do? As parents, grandparents, aunts, uncles or anyone who has been touched by the innocence and unconditional love of a child, you've been there, if only for a moment. Whether it's watching a movie or hearing a tragic story, you can't help but mentally put yourself in that position and consider, "What would I do if I was faced with the threat of losing that precious child?"
Don't be ashamed if you just as quickly dismiss it preferring not to give it any thought. It's just too horrendous. Naturally no one is meant to outlive the children in their life. The answer is obvious. Like you, we chose to fight back.
Sanfilippo Syndrome has been a dark cloud overshadowing our family. We have bravely battled this real life boogie man. Some days we win and some we are left feeling defeated.
The phone call on March 1, 2017 definitely has us feeling like we are heading to a monumental victory. Michaela has been invited to participate in a clinical trial at Columbia University Medical Centre which is scheduled to begin next month. The treatment will require weekly IV infusions for 6 consecutive months. At this point, it is not a cure, but it has the potential to greatly improve her quality of life or perhaps even reverse some symptoms. All medical costs will be covered however, we are responsible for travel expenses, roughly estimated at $25,000 US. Once again, we ask what would you do if it were your child? We find ourselves at the mercy of our community and the kindness of strangers begging for your help and support. We can't possibly do it alone.
Short Version: Michaela’s time is running out. Many children lose their battle with Sanfilippo Syndrome in their early to mid-teens. Michaela continues to fight and we are so grateful that she is beating the odds. She will be 17 in August. The reality of this Boogie Man, that has haunted us for more than a dozen years, is closing in on us. We have a renewed hope and another chance to carry on this fight BUT WE NEED HELP.
Originally, Michaela was invited to participate in a clinical trial at Columbia University Medical Centre, Manhattan, New York that was scheduled to begin in May 2017. Due to money matters, corporate support of this trial was canceled. Lucky for us, TEAM SANFILIPPO stepped in to revive our hope and to make this clinical trial a reality.
In spring 2021, they began treating patients in New York with favourable results. We are looking forward to Michaela starting in 2021 at an upper Michigan site. This treatment has the potential to greatly improve Michaela's quality of life or perhaps even reverse some of the damage caused by Sanfilippo Syndrome. Most medical costs are covered but travel expenses are not. The trial itself has been extended to a 12 month study of WEEKLY infusions. All of the travel expenses are our responsibility.
I find myself overwhelmed by mixed emotions. I’m daring to HOPE for a better life for Michaela. I'm EXHAUSTED by the 13 long years that we've already fought this war knowing full well that the toughest battles remain ahead of us. I'm GRATEFUL for all the love and support throughout these years from our community. I am HUMBLED to be here again, begging for your help and FINANCIAL SUPPORT.
Our Story can be found HERE    .
The ORIGINAL DETAILS: On March 1, 2017, the house phone rang at a very early hour which startled me out of a deep sleep and immediately, that driving force kicked in. You know the one that bolsters you into fight or flight mode because instinct tells you that this call is going to change your life in some important way. Good or bad, it will be a call to action. Thankfully, this was a good news call, one that we had waited more than 8 years to get.
To completely understand its meaning, you have to go back in time to a very different phone call that we received in September of 2008, shortly after Michaela's fourth birthday. It was a call that takes your breath away and leaves you stunned, completely dumbfounded as you try to comprehend the message, frowning, shaking your head, unbelievable. There must be some mistake. Three years of questioning doctor after doctor and a summer of submitting this precious baby girl to poking, prodding and every test they could think of came to an end on that day, we had been searching for an answer and here it was. Michaela has mucopolysaccharidoses (MPS) type IIIB also known as Sanfilippo Syndrome. It is a very rare genetic disorder in which one tiny enzyme is mutated and therefore cannot break down a long chain sugar that is naturally manufactured in the body. These molecules then build up and cause progressive damage. With MPS III B, it begins in the central nervous system. Typically, by age 6, effected children lose their language, both the ability to speak and comprehend. By age 10, they are often wheel chair bound. Piece by piece, their abilities fade away leading to dependence on g-tubes and breathing machines until they leave this world sometime in their mid-teens to early twenties.
This was the bomb that devastated our world. How do you process the fact that this is the future of your beautiful child? After the initial shock wears off, fight or flight kicks in and questions take form. What do we do about this? How do we fight back? How do we save our baby? Who will help us? We were told. "There's nothing you can do. There's no cure. There aren't even any treatments. Just take her home and enjoy her while you can. She's not bad right now but it's coming."
So what would you do? As parents, grandparents, aunts, uncles or anyone who has been touched by the innocence and unconditional love of a child, you've been there, if only for a moment. Whether it's watching a movie or hearing a tragic story, you can't help but mentally put yourself in that position and consider, "What would I do if I was faced with the threat of losing that precious child?"
Don't be ashamed if you just as quickly dismiss it preferring not to give it any thought. It's just too horrendous. Naturally no one is meant to outlive the children in their life. The answer is obvious. Like you, we chose to fight back.
Sanfilippo Syndrome has been a dark cloud overshadowing our family. We have bravely battled this real life boogie man. Some days we win and some we are left feeling defeated.
The phone call on March 1, 2017 definitely has us feeling like we are heading to a monumental victory. Michaela has been invited to participate in a clinical trial at Columbia University Medical Centre which is scheduled to begin next month. The treatment will require weekly IV infusions for 6 consecutive months. At this point, it is not a cure, but it has the potential to greatly improve her quality of life or perhaps even reverse some symptoms. All medical costs will be covered however, we are responsible for travel expenses, roughly estimated at $25,000 US. Once again, we ask what would you do if it were your child? We find ourselves at the mercy of our community and the kindness of strangers begging for your help and support. We can't possibly do it alone.
Organizer
Ann Morris Kameka
Organizer
Windsor, ON
