Pilot Program of Newborn Screening for 5q Spinal Muscular Atrophy in the Russian Federation
5q spinal muscular atrophy (5q SMA) is one of the most common autosomal recessive disorders in the Russian Federation. The first medication to treat 5q SMA was registered in the Russian Federation for treatment of all 5q SMA types in 2019, and the last of the three currently available in December 20...
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MDPI AG,
2023-05-01T00:00:00Z.
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