carbaglu
recordati rare diseases - carglumic acid - amino acid metabolism, inborn errors; propionic acidemia - Önnur meltingarvegi og efnaskipti vörur, - hjá er ætlað í meðferð:hyperammonaemia vegna n-acetylglutamate-þétti aðal skort;hyperammonaemia vegna isovaleric skal;hyperammonaemia vegna methymalonic skal;hyperammonaemia vegna skal propionic.
imbruvica
janssen-cilag international nv - ibrutinib - lymphoma, mantle-cell; leukemia, lymphocytic, chronic, b-cell - antineoplastic agents, protein kinase inhibitors - imbruvica as a single agent is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (mcl). imbruvica as a single agent or in combination with rituximab or obinutuzumab or venetoclax is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (cll) (see section 5. imbruvica as a single agent or in combination with bendamustine and rituximab (br) is indicated for the treatment of adult patients with cll who have received at least one prior therapy. imbruvica as a single agent is indicated for the treatment of adult patients with waldenström’s macroglobulinaemia (wm) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo immunotherapy. imbruvica in combination with rituximab is indicated for the treatment of adult patients with wm.
chenodeoxycholic acid leadiant (previously known as chenodeoxycholic acid sigma-tau)
leadiant gmbh - chenodeoxycholic sýru - xanthomatosis, cerebrotendinous; metabolism, inborn errors - galla og lifrarmeðferð - chenodeoxycholic sýru er ætlað fyrir meðferð meðfæddan villur af aðal galli sýru myndun vegna steról 27 hýdroxýlkljúfs skort (kynna eins og cerebrotendinous xanthomatosis (ctx)) í börn, börn og unglingar á aldrinum 1 mánuð til 18 ára og fullorðnir.
orphacol
theravia - cholic sýru - digestive system diseases; metabolism, inborn errors - galli sýra og afleiður - orphacol er ætlað fyrir meðferð meðfæddan villur í aðal galli-sýru myndun vegna 3β-hýdroxý-Δ5-c27-stera oxidoreductase skort eða Δ4-3-oxosteroid-5β-redúktasa skort á börn, börn og unglingar á aldrinum einn mánuð til 18 ára og fullorðnir.
ucedane
eurocept international bv - carglumic acid - hyperammonemia; amino acid metabolism, inborn errors - Önnur meltingarvegi og efnaskipti vörur, - 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.
vazkepa
amarin pharmaceuticals ireland limited - icosapent ethyl - dýrarannsóknir - lipid breytandi lyf - indicated to reduce cardiovascular risk as an adjunct to statin therapy.
xenpozyme
sanofi b.v. - olipudase alfa - acid sphingomyelinase deficiency (asmd) type a/b or type b - Önnur meltingarvegi og efnaskipti vörur, - 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.
nulibry
tmc pharma (eu) limited - fosdenopterin hydrobromide dihydrate - metal metabolism, inborn errors - Önnur meltingarvegi og efnaskipti vörur, - nulibry is indicated for the treatment of patients with molybdenum cofactor deficiency (mocd) type a.
holoxan innrennslisstofn, lausn 500 mg
baxter medical ab* - ifosfamidum inn - innrennslisstofn, lausn - 500 mg
holoxan innrennslisstofn, lausn 2000 mg
baxter medical ab* - ifosfamidum inn - innrennslisstofn, lausn - 2000 mg