Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases

Objective: To describe a risk-sharing program's implementation and results on enzyme replacement therapy for lysosomal diseases. Method: The program was designed and implemented in a referral hospital for congenital metabolic diseases. The conclusion of agreements required the following phases:...

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Hoofdauteurs: María Dolores Edo-Solsona (Auteur), José Luis Poveda-Andrés (Auteur), Isidro Vitoria-Miñana (Auteur)
Formaat: Boek
Gepubliceerd in: Elsevier, 2020-01-01T00:00:00Z.
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