Orchard Therapeutics Celebrates Addition of Metachromatic Leukodystrophy to the U.S. Recommended Uniform Screening Panel

Core Insights - The addition of metachromatic leukodystrophy (MLD) to the U.S. Recommended Uniform Screening Panel (RUSP) is a significant advancement for newborn screening, enabling early diagnosis and treatment of this severe disease [1][3][5] - Newborn screening (NBS) is recognized as one of the most successful public health programs, with approximately 1 in 500 newborns diagnosed with conditions through NBS, potentially saving over 8,000 infants annually [2][3] - Community-led efforts are crucial for the implementation of newborn screening for MLD, with 14 states already having RUSP-aligned legislation [1][2][8] Company Insights - Orchard Therapeutics, a Kyowa Kirin company, is focused on advancing gene therapy treatments, including the recently approved therapy for MLD, which is the first of its kind for eligible children [5][11] - The company emphasizes the importance of collaboration with researchers, physicians, and patient advocates to enhance and modernize newborn screening programs [3][11] - Orchard Therapeutics aims to leverage its expertise in gene therapy to address severe diseases with high unmet medical needs, including MLD [11][12] Industry Insights - MLD is an ultra-rare neurometabolic disease affecting approximately 1 in 100,000 live births, leading to severe neurological damage and high mortality rates [3][10] - The global recognition of the need for newborn screening for MLD is growing, with several countries, including Norway and Sweden, advancing their screening programs [9] - The successful implementation of newborn screening for MLD in the U.S. is expected to influence other countries to adopt similar measures, highlighting the importance of international collaboration in public health initiatives [8][9]