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

orkambi

vertex pharmaceuticals (ireland) limited - lumacaftor, ivacaftor - cisztás fibrózis - egyéb légzőszervi termékek - orkambi tabletta javallott a cisztás fibrózis (cf) a betegek éves 6 éves vagy idősebb, akik homozigóta a f508del mutáció a cftr gén. orkambi granules are indicated for the treatment of cystic fibrosis (cf) in children aged 1 year and older who are homozygous for the f508del mutation in the cftr gene.

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

kalydeco

vertex pharmaceuticals (ireland) limited - ivacaftor - cisztás fibrózis - egyéb légzőszervi termékek - kalydeco tablets are indicated:as monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (cf) who have an r117h cftr mutation or one of the following gating (class iii) mutations in the cystic fibrosis transmembrane conductance regulator (cftr) gene: g551d, g1244e, g1349d, g178r, g551s, s1251n, s1255p, s549n or s549r (see sections 4. 4 és 5. in a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (cf) who are homozygous for the f508del mutation or who are heterozygous for the f508del mutation and have one of the following mutations in the cftr gene: p67l, r117c, l206w, r352q, a455e, d579g, 711+3a→g, s945l, s977f, r1070w, d1152h, 2789+5g→a, 3272 26a→g, and 3849+10kbc→t. in a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (cf) who have at least one f508del mutation in the cftr gene (see section 5. kalydeco granules are indicated for the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (cf) who have an r117h cftr mutation or one of the following gating (class iii) mutations in the cftr gene: g551d, g1244e, g1349d, g178r, g551s, s1251n, s1255p, s549n or s549r (see sections 4. 4 és 5. in a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (cf) in paediatric patients aged 2 to less than 6 years who have at least one f508del mutation in the cftr gene.

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

vimizim

biomarin international limited - recombinant human n-acetylgalactosamine-6-sulfatase - mucopolysaccharidosis iv - egyéb táplálkozási traktus pedig anyagcsere termékek, - a vimizim iva típusú (morquio a szindróma, mps iva) mucopolysaccharidosis kezelésére javallt minden korosztályban.

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

brineura

biomarin international limited - az alfa cerliponáz - neuronális ceroid-lipofuscinosisok - egyéb táplálkozási traktus pedig anyagcsere termékek, - brineura kezelésére javallt, a neuronális ceroid lipofuscinosis 2-es típusú (cln2) betegség, vagy más néven tripeptidyl peptidase 1 (tpp1) hiány,.