For the intractable disease "mucopolysaccharidosis" that causes intellectual developmental delay in children, the world's first treatment method to suppress the progression of symptoms has been approved and is expected to be put into practical use on the 26th.
The treatment method was developed by a group of directors of the National Center for Child Health and Development, Torayuki Okuyama.
"Mucopolysaccharidosis type 2" is an intractable disease of children that is born with a lack of specific enzymes and causes intellectual developmental delay and organ damage, and it is estimated that there are at least about 150 patients in Japan.
By administering enzymes into the body, the progression of symptoms can be suppressed to a certain extent, but until now it has not been possible to administer it to the brain, and it has not been possible to suppress the delay in intellectual development.
Therefore, in a clinical trial, the research group implanted a special device under the scalp of six severely ill children and administered the enzyme directly into the brain once a month for three years.
As a result, the progression of symptoms was suppressed in children who started administration before the age of three, and the Ministry of Health, Labor and Welfare approved it as a treatment method.
The world's first treatment method is expected to be put into practical use from the 26th, and it costs about 4 million yen per administration, but if you use multiple subsidy programs, you will have to pay a monthly out-of-pocket amount. It means that it can be suppressed to less than 10,000 yen.
The parents of a 7-year-old boy who participated in the trial said, "Until now, I could only see it getting worse, but now I can say my name and have hope for the future." I was talking.