Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, has announced a number of updates pertaining to the advancement of universal newborn screening (NBS) for metachromatic leukodystrophy (MLD).
NBS is widely considered one of the most successful public health programs worldwide. In the U.S., approximately 1 in 500 newborns have a condition that can be diagnosed through NBS, and more than 8,000 infants have the potential to receive life-saving treatment due to this public health program annually.
MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease that affects approximately one in 100,000 live births. It is caused by an error in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. These children eventually deteriorate into a vegetative state, which may require 24-hour intensive care, and the majority pass away within five years of symptom onset, creating an enormous emotional and financial burden on the family.
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