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Announced in June 2025, the CNPV program aims to expedite the review of drugs that address unmet medical needs, improve affordability, or support public health priorities

The absence or diminished function of ATP7B results in accumulation of copper in the hepatocyte due to both impaired copper excretion into bile and inability to incorporate copper into ceruloplasmin.14 The production of ceruloplasmin without attached copper (apoceruloplasmin), which has a shorter half-life than ceruloplasmin, results in decreased levels of ceruloplasmin in the blood of Wilsons patients

Retrieved from: Lombard, K.A

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