New gene therapy reimbursed for children with MLD - UMC Utrecht

Description

New gene therapy is reimbursed for children with a very rare metabolic disease (MLD). MLD is a severe genetic disease caused by a mistake in DNA. This gene therapy is the only effective treatment for a severe variant of MLD in which children deteriorate rapidly. The Dutch government recently started reimbursing this therapy for patients who have no symptoms yet.

Period	Jan 2024
Held at	Child Health
Degree of Recognition	International