At the age of six, Loïc, who suffers from Morquio disease, replied to a child who was staring at him one day in a shop: “I’m just a little more different than you. That’s right, like other kids, he plays field hockey, baseball, ping pong and karate!
Loïc is taking part in a clinical study at the Montreal Children’s Hospital in preparation for a first treatment developed by the Biomarin company. Hope for people suffering from this disease.
Morquio disease or syndrome
Mucopolysaccharidosis type IV (two forms: A and B)
MPS-IV
Morquio disease is an inherited, progressive disorder belonging to a class of diseases known as “lysosomal overload”. Glycoaminoglycans or mucopolysaccharides (large sugar molecules), which are normally eliminated or recycled by the body, accumulate in the body’s cells. Symptoms appear during childhood as a result of this accumulation, and present themselves as abnormal bone development, generally resulting in significant physical handicap and short stature, without intellectual deficits. Other body systems may also be affected (cardiac, pulmonary, auditory, etc.). The disease can vary greatly from one person to another.
Morquio disease affects boys and girls equally. Approximately one person in 30,000 is affected, born to parents who do not have the disease (they carry the defective gene, but are not affected). Two carrier parents have a 25% probability of having an affected child in each pregnancy.