Xenpozyme สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

xenpozyme

sanofi b.v. - olipudase alfa - acid sphingomyelinase deficiency (asmd) type a/b or type b - drugi gastrointestinalni trakt i metabolizam, lijekovi, - xenpozyme is indicated as an enzyme replacement therapy for the treatment of non-central nervous system (cns) manifestations of acid sphingomyelinase deficiency (asmd) in paediatric and adult patients with type a/b or type b.

Granupas (previously Para-aminosalicylic acid Lucane) สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

granupas (previously para-aminosalicylic acid lucane)

eurocept international b. v. - par-аминосалициловая kiselina - tuberkuloza - antimikobakterija - granupas je indiciran za uporabu kao dio odgovarajući kombiniranom za multi-rezistentna tuberkuloza u odraslih i pedijatrijskih bolesnika od 28 dana starosti i starije kada učinkovite terapije ne može inače biti sastoji iz razloga otpor ili podnošljivost (vidjeti dio 4.. treba uzeti u obzir formalne preporuke za odgovarajuće korištenje antibakterijskih posrednika.

Orphacol สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

orphacol

theravia - kolesku kiselinu - digestive system diseases; metabolism, inborn errors - Žučnih kiselina i derivata - orphacol je indiciran za liječenje urođene pogreške u primarne žučne kiseline sinteza 3β-hidroksi-Δ5-c27-steroidni oxidoreductase manjak ili nedostatak Δ4-3-oxosteroid-5β-reduktaze u dojenčadi, djece i adolescenata u dobi od jednog mjeseca do 18 godina i odrasle osobe.

Givlaari สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

givlaari

alnylam netherlands b.v. - givosiran - Порфирий, zatajenje - različite dijelove probavnog trakta i metabolizma proizvodi - liječenje akutnog zatajenja порфирии (АХА) kod odraslih i adolescenata u dobi od 12 godina i stariji.

Carbaglu สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

carbaglu

recordati rare diseases - karglumna kiselina - amino acid metabolism, inborn errors; propionic acidemia - drugi gastrointestinalni trakt i metabolizam, lijekovi, - carbaglu je indiciran u liječenju:hyperammonaemia komunikacije do n-acetylglutamate-sintaze primarni deficit;hyperammonaemia zbog изовалериановой acidaemia;hyperammonaemia zbog methymalonic acidaemia;hyperammonaemia zbog пропионовой acidaemia.

Ucedane สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

ucedane

eurocept international bv - karglumna kiselina - hyperammonemia; amino acid metabolism, inborn errors - drugi gastrointestinalni trakt i metabolizam, lijekovi, - ucedane is indicated in treatment of:hyperammonaemia due to n-acetylglutamate synthase primary deficiency;hyperammonaemia due to isovaleric acidaemia;hyperammonaemia due to methymalonic acidaemia;hyperammonaemia due to propionic acidaemia.

Vizimpro สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

vizimpro

pfizer europe ma eeig - dacomitinib monohidrat - karcinom, ne-malih stanica pluća - antineoplastična sredstva - vizimpro, kao monoterapija, je propisan za prva linija terapije kod odraslih pacijenata sa lokalno-održao ili метастатического немелкоклеточного raka pluća (НМРЛ) s epidermalnog faktora rasta (egfr) aktiviraju mutacije.

Hemgenix สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

hemgenix

csl behring gmbh - etranacogene dezaparvovec - hemofilija b - other hematological agents - treatment of severe and moderately severe haemophilia b (congenital factor ix deficiency) in adult patients without a history of factor ix inhibitors.

Libmeldy สหภาพยุโรป - โครเอเชีย - EMA (European Medicines Agency)

libmeldy

orchard therapeutics (netherlands) bv - atidarsagene autotemcel - leukodystrophy, metachromatic - ostali lijekovi protiv živčanog sustava - libmeldy is indicated for the treatment of metachromatic leukodystrophy (mld) characterized by biallelic mutations in the arysulfatase a (arsa) gene leading to a reduction of the arsa enzymatic activity:in children with late infantile or early juvenile forms, without clinical manifestations of the disease,in children with the early juvenile form, with early clinical manifestations of the disease, who still  have the ability to walk independently and before the onset of cognitive decline.