Hi, My name is Pia Boehnke. I am a super cheerful girl of 8 months old (27. Nov 2018). I mainly use my eyes to discover the world, and I take everything in. What I enjoy most is watching my brother Briek go crazy and cuddling with my mom and dad.
But sometimes it really frustrates me that I can’t use my arms and legs properly and that I can’t hold all my fun toys. I also can’t sit up straight like other kids my age.
WHAT DO I HAVE?
At 4 months old, the doctor diagnosed me with SMA Type 1, Spinal Muscular Atrophy. SMA is an incurable genetic muscle disease. The earlier it is diagnosed, the worse the verdict. I am missing the SMN-1 gene that helps me fully use my muscles. If I was diagnosed with this disease two years ago, I would have died before the age of two. Fortunately they invented a medicine, Spinraza®, that will keep me alive and helps me to use my arms and hands and my legs and feet a little bit. But I will never be able to crawl, let alone walk.
Unless a miracle happens…
WHAT DO I NEED?
Recently, this miracle happened! A wonderous medicine was invented, called Zolgensma. It is a gene-therapy, administered in one shot, that will put the SMN-1 gene I’m missing into my DNA. The first results of this medicine are very promising. I could lead a long, beautiful life, in which I might even learn to walk, or at least be more independent.
There is only one problem, one big problem. The medicine I need is the most expensive one in the world, at almost 2 million Euros. Unfortunately, my mom and dad can’t afford that. The company that makes the medicine, Novartis, won’t give it to me for free and I was diagnosed two weeks too late too be in a clinical trial. I’m currently the only child in Belgium not in such a trial, and thus the only one that doesn’t get this medicine. My mom and dad tried everything to get the medicine from the company, but Novartis wouldn’t hear of it. Moreover, it is of the essence that I get this medicine as soon as possible, because the sooner I receive it, the more progress I will make. Unfortunately it’s also not option to wait for an agreement to reimburse the medicine in Belgium, because it wil likely take almost a year before there will be an agremeent and it Novartis has already claimed it will only seek reimbursement for children until the age of 6 months.
WILL YOU HELP ME?
My mom and dad, though, are not willing to give up. They will do anything it takes to collect the money needed for my miracle-medicine. They want to set up a big charity event to collect as much money as possible.
Are you a performer that wants to give a show here, a mom or dad that wants to help by spending a few coins, or just someone with two hands that wants to help us set up this event. You would be most welcome, because a lot of smalls will make a big one too.
Are you interested? You can reach me at firstname.lastname@example.org or deposit a gift on my account BE05 3631 9038 5475 (BIC: BBRUBEBB).
I will promise you to be a strong, sweet girl and I will fight as hard as I can!