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Genetic landscape of phenylketonuria in Brazil
Jul 9, 2026
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Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases
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Integrative and systematic genomic approaches to improve diagnosis in rare and undiagnosed diseases: results from the RareBoost project
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Sustained clinical benefit of idursulfase beta in mucopolysaccharidosis II: two-year experience from a phase 3 extension study including patients switched from idursulfase
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