PHARMAC's Rare Disorder Subcommittee

The Subcommittee met on 5 and 6 November 2018 and has recommended that four medicines be funded, and five be declined for funding. One medicine was deferred to be considered at a later date.

The recommendations were that the four medicines be funded for use in hyperammonaemia (with two different patient groups), hereditary tyrosinaemia type 1, Fabry disease, and cystic fibrosis in patients with a G551D mutation.

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