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Table 3 Categories of EMA-approved OMPs for HMDs according to accessibility and delivery to patients in the MetabERN centres

From: Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network

OMP accessible in > 2/3 of the centres
(mean = 88.5%)
OMP accessible in < 2/3 of the centres
(mean = 40.2%)
Delivered to >  1/3 of the patients
(mean = 70.1%, range 38–100%)
Delivered to <  1/3 of the patients
(mean = 15.8% range 7–23%)
Delivered to >  1/3 of the patients
(mean = 66.3% range 45–100%)
Delivered to <  1/3 of the patients
(mean = 6.0% range 0–18%)
Sodium PhenylbutyrateCarglumic acidChenodeoxycholic acid •Glycerol phenylbutyrate
Nitisinone •SapropterinCysteamine hydrochloride 
MiglustatMigalastatCysteamine bitartrateAlipogene tiparvovec
Agalsidase alphaEliglustatCerliponaseADA CD34 cells
Agalsidase betaVelagluceraseElosulfase 
Imiglucerase Sebelipase 
Alglucosidase alpha * Afamelanotide 
Laronidase   
Idursulfase •   
Galsulfase •   
  1. Information about the number of patients followed in the centres is missing for Cholic acid (accessible in more than 2/3 of the centres), Idebenone (accessible in less than 2/3 of the centres) and Asfotase alpha (accessible in less than 2/3 of the centres). • indicates products prescribed to almost all the patients with the considered condition