Sapropterin Dipharma Európai Unió - magyar - EMA (European Medicines Agency)

sapropterin dipharma

dipharma arzneimittel gmbh - szapropterin dihidroklorid - phenylketonurias - egyéb táplálkozási traktus pedig anyagcsere termékek, - sapropterin dipharma is indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with phenylketonuria (pku) who have been shown to be responsive to such treatment. sapropterin dipharma is also indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with tetrahydrobiopterin (bh4) deficiency who have been shown to be responsive to such treatment.

Nulibry Európai Unió - magyar - EMA (European Medicines Agency)

nulibry

tmc pharma (eu) limited - fosdenopterin hydrobromide dihydrate - metal metabolism, inborn errors - egyéb táplálkozási traktus pedig anyagcsere termékek, - nulibry is indicated for the treatment of patients with molybdenum cofactor deficiency (mocd) type a.

Opfolda Európai Unió - magyar - EMA (European Medicines Agency)

opfolda

amicus therapeutics europe limited - miglustat - ii típusú glikogén-tároló betegség - egyéb tápcsatorna és metabolizmus termékek - opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset pompe disease (acid α- glucosidase [gaa] deficiency).