Europeiska unionen - svenska - EMA (European Medicines Agency)

orchard therapeutics (netherlands) bv - atidarsagene autotemcel - leukodystrophy, metachromatic - andra nervsystemet droger - 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.

Europeiska unionen - svenska - EMA (European Medicines Agency)

bluebird bio (netherlands) b.v. - elivaldogene autotemcel - adrenoleukodystrophy - andra nervsystemet droger - treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an abcd1 genetic mutation, and for whom a human leukocyte antigen (hla) matched sibling haematopoietic stem cell donor is not available.

Europeiska unionen - svenska - EMA (European Medicines Agency)

roche registration gmbh - satralizumab - neuromyelitis optica - immunsuppressiva - satralizumab (enspryng) is indicated as a monotherapy or in combination with immunosuppressive therapy (ist) for the treatment of neuromyelitis optica spectrum disorders (nmosd) in adult and adolescent patients from 12 years of age who are anti-aquaporin-4 igg (aqp4-igg) seropositive.

Europeiska unionen - svenska - EMA (European Medicines Agency)

biomarin international limited - vosoritide - achondroplasia - läkemedel för behandling av bensjukdomar - voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. the diagnosis of achondroplasia should be confirmed by appropriate genetic testing.

Europeiska unionen - svenska - EMA (European Medicines Agency)

amryt pharmaceuticals dac - octreotide acetate - akromegali - hypofysiska och hypotalamiska hormoner och analoger - mycapssa is indicated for maintenance treatment in adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues.

Europeiska unionen - svenska - EMA (European Medicines Agency)

united therapeutics europe ltd - dinutuximab - neuroblastom - antineoplastiska medel - unituxin är indicerat för behandling av högrisk neuroblastom hos patienter i åldern 12månader till 17år, som tidigare har mottagit induktionskemoterapi och uppnått minst ett delsvar, följt av myeloablativ terapi och autolog stamcellstransplantation transplantation (asct). det administreras i kombination med granulocyt-makrofagkolonistimulerande faktor (gm-csf), interleukin-2 (il-2) och isotretinoin.

Sverige - svenska - myHealthbox

bayer ag - acetylsalicylsyra - tablett - 500 mg - acetylsalicylsyra 500 mg aktiv substans - acetylsalicylsyra

Europeiska unionen - svenska - EMA (European Medicines Agency)

retrophin europe ltd - kolsyra - metabolism, infödda fel - gall- och leverterapi - cholsyra fgk är indicerat för behandling av medfödda fel av primära gallsyror syntes, till spädbarn från en månads ålder för kontinuerlig livslång behandling genom vuxen ålder, som omfattar följande enda enzym fel:sterol 27-hydroxylas (presentation som cerebrotendinous xanthomatosis, ctx) brist;2- (eller alpha-) methylacyl-coa racemase (amacr) brist. kolesterol 7 alfa-hydroxylas (cyp7a1) brist.

Sverige - svenska - Läkemedelsverket (Medical Products Agency)

meda ab - acetylsalicylsyra - tablett - 500 mg - acetylsalicylsyra 500 mg aktiv substans