Posts for Stewarding Entity: Therapeutics Committee
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Olipudase alfa approved for pediatric and adult patients with acid sphingomyelinase deficiency: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
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Vosoritide approved for treatment of linear growth in pediatric patients with achondroplasia: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
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Phenylalanine hydroxylase deficiency treatment and management: A systematic evidence review of the American College of Medical Genetics and Genomics (ACMG)
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Solid organ transplantation in methylmalonic acidemia and propionic acidemia: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG)
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Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG)
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Phenylalanine hydroxylase deficiency: diagnosis and management guideline
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Phenylalanine hydroxylase deficiency: diagnosis and management guideline (French)
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Phenylalanine hydroxylase deficiency: diagnosis and management guideline (German)
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Phenylalanine hydroxylase deficiency: diagnosis and management guideline (Italian)
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Phenylalanine hydroxylase deficiency: diagnosis and management guideline (Portuguese)