Metachromatic Leukodystrophy (MLD)
This article reviews the agreement made by Orchard Therapeutics, allowing the usage of the drug Libmeldy for treating Metachromatic Leukodystrophy (MLD) in Germany. Orchard Therapeutics is a global industry responsible for generating gene therapies for rare diseases such as MLD. Specifically, MLD is a genetically inherited disease affecting tissues and organs such as the brain, liver, gallbladder, kidneys, and spleen. The underlying cause of the condition is the accumulation of sulfatides due to mutations in the arylsulfatase-A (ARSA) gene. Sulfatides are a subtype of lipids responsible for the correct function of the nervous system. Therefore, their accumulation interferes with the normal role of the neurons leading to neurological problems such as the inability to walk, see, eat, and swallow. By the age of five, the mortality rate is over 50% enabling the search for a treatment an urgent need. Agreement between companies allowed the free screening of new-borns in Germany and other European countries allowing early diagnosis and possible treatment with the newly approved drug, Libmeldy. This drug acts by reducing the effects of the impaired ARSA protein in patients with early onset symptoms. As a result, Libmeldy is approved in various countries including Germany, allowing the successful treatment of patients, giving them the chance of having a better life quality.