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EC grants orphan drug status to JR-446 for MPS IIIB

Summary by Pharmaceutical Business Review
This follows a similar endorsement by the US Food and Drug Administration (FDA). Sanfilippo syndrome type B is an autosomal recessive disease marked by mutations in the N-acetyl-alpha-glucosaminidase The post EC grants orphan drug status to JR-446 for MPS IIIB appeared first on Pharmaceutical Business review.
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Pharmaceutical Business review broke the news in on Wednesday, June 25, 2025.
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