New Treatment For Mucopolysaccharidosis Available
A treatment for children with mucopolysaccharidosis II, also known as Hunter syndrome, will be available for the first time in the world when it launches in Japan on Monday.
MPS II is caused by an enzyme deficiency and affects a child's physical and mental development. Estimates put the number of sufferers in Japan at 150 or more.
Giving the enzymes to patients can help to decelerate the progress of the disease, but it had not been possible to administer them into brain and suppress its delay of brain development.
Dr. Okuyama Torayuki from the National Center for Child Health and Development and his group have come up with a treatment to slow down the progress of the disease.
The new treatment uses a special apparatus to deliver the specific enzyme to the brain.
The treatment can cost about 40,000 dollars for each administration of the enzyme. But various assistance and subsidies can bring down the monthly cost for a patient to around several hundred dollars.
The parents of a 7-year-old boy who took part in the clinical trial said that they could do nothing but watch his condition deteriorate. They said now, he is getting better and can now say his own name.