Kamal Hoteit is a ten year old boy who suffers from a rare life limiting condition known as Morquio Syndrome. This is a life limiting inherited disease that affects one in every 250,000 people.
Kamal was diagnosed with Morquio Syndrome at the age of two. He suffers with joint and muscle pain, as well as back pain caused by Kyphoscoliosis. He also has vision and hearing problems. Kamal has a short stature, but his internal organs are still growing at normal rate, so his breathing can be restricted. He has had several operations in his life time: ear grommets inserted several times to help with his glue ear, removal of his adenoids and tonsils, a cervical neck fusion and decompression of the spine. He has had two operations to insert eight plates in his knees and one operation to insert plates into his ankles.
Kamal has been trialling the drug Vimizim, since July 2009, and since February this year has been receiving this drug at home with a health care nurse.
Kamal had his neck fusion at the age of three and a half years old. The surgeons had explained that he will require his Kyphoscoliosis corrected within a year’s time but since being on the drug his Kyphoscolisis has not caused any concern for any surgery to take place as the surgeons at Great Ormond Street Hospital are happy that at the moment his spine is stable without any operations. However, I believe that if the drug was to stop then Kamal would require his spinal surgery which would require him to take a long time to recover as well as cause him excruciating pain.
Before taking the drug, Kamal would easily get tired. He would require a two to three hour nap when he would come home from school. Since being on the drug Kamal is able to take part in more activities and have more energy and is able to try and enjoy life like any other normal ten year old boy. Not only does he have more energy it has benefited him medically too.
Since being on the drug Kamal’s lung function has improved significantly and Dr Wallis at Great Ormond Street Hospital is amazed by Kamal’s improvement since being on Vimizim. He has even grown a few centimetres since he began the trial, and within the last year alone he has grown one and a half centimetres. I know this may seem like nothing compared to a child his age, but with a child with Morquio Syndrome any millimetre of growth is a benefit.
If funding is not granted then any pre-existing damage will not be repaired and new damage would begin. I have seen such an improvement in Kamal and it would be heart breaking to see him deteriorate. Kamal’s only wish for Father Christmas is ‘to be a normal boy’ and by having Vimizim it gives him the chance to be as normal as can be.