New gene therapy could save the lives of children with a severe inherited disease

The gene therapy for MLD involves modifying stem cells in a laboratory in Italy before they are reintroduced to the patient, says Erik Eklund.
The rare genetic disease MLD primarily affects younger children and, until recently, has resulted in significant suffering and a drastically shortened lifespan. Now, there is a life-saving treatment, and Skåne University Hospital is the first hospital in the Nordic region to treat children with this severe illness.

"It's an incredible feeling to be part of providing families with hope for a cure when there was no solution before," says Erik Eklund, Senior Consultant in paediatric neurology at Skåne University Hospital and associate professor in experimental paediatrics at Lund University.

He is part of the medical and nursing team currently treating a child with the rare neurological disease Metachromatic Leukodystrophy (MLD), which typically affects younger children and leads to the loss of the ability to walk and swallow, along with impaired cognitive functions.

"The course and severity of the disease depend on the age at which symptoms appear. But until now, it has always resulted in a significantly shortened lifespan," says Erik Eklund.