European Union - English - EMA (European Medicines Agency)

recordati rare diseases - zinc - hepatolenticular degeneration - other alimentary tract and metabolism products, - treatment of wilson's disease.

European Union - English - EMA (European Medicines Agency)

amicus therapeutics europe limited - migalastat hydrochloride - fabry disease - migalastat - galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of fabry disease (α-galactosidase a deficiency) and who have an amenable mutation.

European Union - English - EMA (European Medicines Agency)

gmp-orphan sa - trientine tetrahydrochloride - hepatolenticular degeneration - other alimentary tract and metabolism products, - cuprior is indicated for the treatment of wilson's disease in adults, adolescents and children ≥ 5 years intolerant to d-penicillamine therapy.,

Israel - English - Ministry of Health

j-c health care ltd - miglustat - capsules - miglustat 100 mg - miglustat - miglustat - zavesca is indicated for the oral treatment of mild to moderate type i gaucher disease. zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with niemann-pick type c disease.

Israel - English - Ministry of Health

medison pharma ltd - sapropterin dihydrochloride - tablets soluble - sapropterin dihydrochloride 100 mg - sapropterin - sapropterin - kuvan 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.kuvan 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.

European Union - English - EMA (European Medicines Agency)

sanofi b.v. - eliglustat - gaucher disease - other alimentary tract and metabolism products, - cerdelga is indicated for the long-term treatment of adult patients with gaucher disease type 1 (gd1), who are cyp2d6 poor metabolisers (pms), intermediate metabolisers (ims) or extensive metabolisers (ems).

European Union - English - EMA (European Medicines Agency)

biomarin international limited - sapropterin dihydrochloride - phenylketonurias - other alimentary tract and metabolism products, - kuvan 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.kuvan 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.

European Union - English - EMA (European Medicines Agency)

takeda pharmaceuticals international ag ireland branch - teduglutide - malabsorption syndromes - other alimentary tract and metabolism products, - revestive is indicated for the treatment of patients aged 1 year and above with short bowel syndrome (sbs). patients should be stable following a period of intestinal adaptation after surgery.revestive is indicated for the treatment of patients aged 1 year and above with short bowel syndrome. patients should be stable following a period of intestinal adaptation after surgery.

European Union - English - EMA (European Medicines Agency)

immedica pharma ab - glycerol phenylbutyrate - urea cycle disorders, inborn - other alimentary tract and metabolism products, - ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (ucds) including deficiencies of carbamoyl phosphate-synthase-i (cps), ornithine carbamoyltransferase (otc), argininosuccinate synthetase (ass), argininosuccinate lyase (asl), arginase i (arg) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (hhh) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

European Union - English - EMA (European Medicines Agency)

piramal critical care b.v. - miglustat - gaucher disease - other alimentary tract and metabolism products, - yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 gaucher disease.yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with niemann-pick type c disease.