19-month-old Teddi Shaw was born with the rare condition metachromatic leukodystrophy (MLD). Last year between June and October she underwent gene therapy. A patient’s faulty gene is replaced by a healthy version of the gene.
“Teddi is doing great,” says the girl’s mother in conversation with English media. “She is walking, running and showing no signs of MLD so far.”
Hereditary
There are currently no treatments available for MLD other than with the drug Libmeldy. The condition is hereditary and causes damage to the nervous system and organs. Children with this condition can move less and less well and develop an intellectual disability. On average, they die around the age of five if they are not treated. Libmeldy can cure children with MLD, provided they do not yet have any symptoms have the disease.
For Teddi’s older sister, who also suffers from MLD, the drug comes too late. Her disease is already far advanced, so she would not benefit from the treatment. In addition to the pain about her older daughter, the mother is grateful that her youngest daughter has perspective again. “Without this treatment, we would lose both children.”
In the Netherlands, this condition is diagnosed in 2 to 3 children per year. The drug Libmeldy is now also on the Dutch market. A spokesperson for the Ministry of Health, Welfare and Sport tells RTL Nieuws that it is not yet reimbursed because negotiations with the manufacturer of the drug are still ongoing.
At the moment, therefore, the most expensive drug in the Netherlands remains Zolgensma, with a price of around 2 million euros per patient. This medicine is given for the muscle disease SMA1.
