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 Phenylbutyrate | Carglumic acid | Chenodeoxycholic acid • | Glycerol phenylbutyrate |
Nitisinone • | Sapropterin | Cysteamine hydrochloride | |
Miglustat | Migalastat | Cysteamine bitartrate | Alipogene tiparvovec |
Agalsidase alpha | Eliglustat | Cerliponase | ADA CD34 cells |
Agalsidase beta | Velaglucerase | Elosulfase | |
Imiglucerase | Sebelipase | ||
Alglucosidase alpha * | Afamelanotide | ||
Laronidase | |||
Idursulfase • | |||
Galsulfase • |