Children in England with a rare and fatal genetic condition will soon be able to get the world’s most expensive drug after NHS bosses negotiated a substantial cut in its £2.8m price.
NHS England hailed the deal that will make the “life-saving” therapy, called Libmeldy, available to the small number of babies and young children who have metachromatic leukodystrophy (MLD). Those who receive it will be able to enjoy a normal life, it said.
The inherited neurodegenerative disorder severely damages a child’s nervous system and organs. Sufferers experience progressive disability similar to that caused by multiple sclerosis. Symptoms include spasticity, seizures and difficulty talking, swallowing, hearing and seeing.
Read more at: https://www.theguardian.com/society/2022/feb/04/children-in-england-with-fatal-condition-to-get-worlds-most-expensive-drug