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Octobre[removed]Listes des médicaments pour les maladies rares en Europe*
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Document Date: 2014-11-03 09:45:01

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City

Philadelphia / /

Company

Shire Orphan Therapies GmbH / Bayer Pharma AG / Mediwound Germany Gmbh / uniQure biopharma B.V. / Eisai Ltd / Genzyme Europe B.V. / Swedish Orphan Biovitrum International AB / Roche Registration Limited / FGK Representative Service GmbH / Gilead Sciences International Limited / Amgen Europe B.V. / Otsuka Novel Products GmbH / Medac GmbH / TMC Pharma Services Ltd. / Farmaceutici SpA / Meda AB / Novartis Europharm Ltd / Glaxo Group Ltd / Pharmaxis Pharmaceuticals Limited / Orphan Europe S.a.r.l. / ARIAD Pharma Ltd / Eaton / Shire Human Genetic Therapies AB / Takeda A/S / Janssen-Cilag International NV / Biomarin Europe Ltd / Merck Serono Europe Ltd / Gentium S.p.a. / InterMune UK Ltd. / Vertex Pharmaceuticals (U.K.) Limited / Actelion Registration Ltd / Celgene Europe Limited / Pfizer Ltd / /

Continent

Europe / /

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Event

Man-Made Disaster / /

Facility

Genzyme Europe B.V. NAGLAZYME N-acetylgalactosamine-4sulfatase INN / /

IndustryTerm

treatment of growth failure / medicinal products / treatment of Lambert-Eaton myasthenic syndrome / treatment of severe hepatic veno-occlusive disease / treatment of patients with Hunter syndrome / treatment of seizures associated with Lennox Gastaut syndrome / le site Internet de / treatment of cystic fibrosis / /

MedicalCondition

delta4-3Oxosteroid-5bêta-reductase deficiency / severe myoclonic epilepsy / rare diseases / seizures / arterial hypertension / blast phase / pulmonary arterial hypertension / metastatic medullary thyroid carcinoma / HAE / Pulmonary / relapsed and refractory multiple myeloma / chronic phase / burns / chronic myeloid leukaemia / phenylketonuria / multi-drug resistant tuberculosis / myelofibrosis / homocystinuria / malnutrition / primary apnea / Hunter syndrome / cerebrotendinous xanthomatosis / hypothyroidism / chronic treatment / hepatocellular carcinoma / lymphoma and multiple myeloma / post-polycythaemia-vera myelofibrosis / thyroid carcinoma / malignant glioma / post-essential-thrombocythaemia myelofibrosis / hereditary tyrosinemia type / pancreatitis / hyperphenylalaninaemia / PKU / chronic lymphocytic leukaemia / deficiency / chronic pulmonary infections / advanced renal cell carcinoma / thrombocytopenic purpura / refractory generalized tonicclonic seizures / T-ALL / severe hepatic veno-occlusive disease / Pseudomonas / Mucopolysaccharidosis II / lymphoblastic lymphoma / corrected simple congenital heart disease / 3bêta-Hydroxy-delta5-C27steroid oxidoreductase deficiency / Dravet’s syndrome / acute myeloid leukaemia / sinusoidal obstructive syndrome / primary myelofibrosis / leukaemia / hereditary angioedema / Maroteaux-Lamy syndrome / C1esterase-inhibitor deficiency / Lennox Gastaut syndrome / cystic fibrosis / high-grade resectable non-metastatic osteosarcoma / Pompe disease / Lambert-Eaton myasthenic syndrome / acid α-glucosidase deficiency / chronic iron / refractory chronic lymphocytic leukaemia / Mucopolysaccharidosis VI / thromboembolic pulmonary hypertension / disease / secondary acute myeloid leukaemia / patent ductus arteriosus / pulmonary multi-drug resistant tuberculosis / Hodgkin lymphoma / N-acetylglutamate synthase primary deficiency / familial lipoprotein lipase deficiency / connective tissue disorders / connective tissue disease / refractory systemic anaplastic large cell lymphoma / /

MedicalTreatment

surgery / surgical treatment / blood transfusions / adjunctive therapy / chemotherapy / enzyme replacement therapy / /

Organization

World Health Organization / /

Person

Lennox Gastaut / /

Position

Hunter / /

Product

ORFADIN / INCRELEX / Cystadane / Ibuprofen / NEXAVAR / NAGLAZYME / DACOGEN / EXJADE / dexamethasone / Orphanet Report Series / Mannitol / KUVAN / MYOZYME / Caffeine citrate / ELAPRASE / /

Technology

stem cells / html / chemotherapy / alpha / transplantation / http / /

URL

http /

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