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Guidance programme

Showing 1 to 2 of 2 results for sapropterin

  1. Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)

    Evidence-based recommendations on sapropterin for treating hyperphenylalaninaemia in phenylketonuria.

  2. HST routing criteria: encouraging innovation in the treatment of ultra-rare diseases

    Our chief medical officer, Professor Jonathan Benger, explains the crucial role the HST programme plays in encouraging innovation in the treatment of ultra-rare diseases. He also highlights why effective routing is essential to getting the best care to patients across the NHS.