Back to Results
First PageMeta Content
Orphan drugs / Piperazines / Lymphocytic leukemia / Anilides / Organofluorides / Chronic myelogenous leukemia / Dasatinib / Ponatinib / Leukemia / Medicine / Health / Oncology


Orphanet Berichtsreihe Orphan Drugs Datenerhebung
Add to Reading List

Document Date: 2013-10-30 07:15:01

Open Document

File Size: 551,15 KB

Share Result on Facebook

City

Philadelphia / /

Company

Shire Orphan Therapies GmbH / Bayer Pharma AG / Mediwound Germany Gmbh / uniQure biopharma B.V. / Eisai Ltd / ViroPharma / Genzyme Europe B.V. / Development Centre (Europe) Ltd / Swedish Orphan Biovitrum International AB / Raptor Pharmaceuticals Europe B.V. / Gilead Sciences International Limited / Amgen Europe B.V. / Medac GmbH / Farmaceutici SpA / Meda AB / Novartis Europharm Ltd / Glaxo Group Ltd / Pharmaxis Pharmaceuticals Limited / Lipomed GmbH / Orphan Europe S.a.r.l. / ARIAD Pharma Ltd / Eaton / Shire Human Genetic Therapies AB / Janssen-Cilag International NV / Biomarin Europe Ltd / SpePharm Holding B.V. / Merck Serono Europe Ltd / InterMune UK Ltd. / Vertex Pharmaceuticals (U.K.) Limited / Celgene Europe Limited / Pfizer Ltd / /

/

Event

Man-Made Disaster / /

Facility

Genzyme Europe B.V. NAGLAZYME N-acetylgalactosamine-4sulfatase INN / Takeda Global Research and Development Centre / /

IndustryTerm

treatment of growth failure / medicinal products / treatment of Lambert-Eaton myasthenic syndrome / treatment of patients with Hunter syndrome / treatment of seizures associated with Lennox Gastaut syndrome / Treatment of adult patients with Short Bowel Syndrome / treatment of cystic fibrosis / /

MedicalCondition

blast phase CML / delta4-3Oxosteroid-5bêta-reductase deficiency / severe myoclonic epilepsy / congenital heart disease / multiple myeloma / seizures / proven nephropathic cystinosis / blast phase / severe / chronic pain / blast crisis / pulmonary arterial hypertension / HAE / chronic phase / relapsed and refractory multiple myeloma / burns / chronic myeloid leukaemia / phenylketonuria / myelofibrosis / adrenal insufficiency / homocystinuria / adrenal cortical carcinoma / primary apnea / Hunter syndrome / primary pulmonary hypertension / hepatocellular carcinoma / lymphoma and multiple myeloma / post-polycythaemia-vera myelofibrosis / malignant glioma / post-essential-thrombocythaemia myelofibrosis / hereditary tyrosinemia type / Short Bowel Syndrome / pancreatitis / hyperphenylalaninaemia / atypical haemolytic uraemic syndrome / PKU / deficiency / renal failure / chronic pulmonary infections / advanced renal cell carcinoma / thrombocytopenic purpura / refractory generalized tonicclonic seizures / T-ALL / nephropathic cystinosis / Pseudomonas / Mucopolysaccharidosis II / lymphoblastic lymphoma / 3bêta-Hydroxy-delta5-C27steroid oxidoreductase deficiency / Dravet’s syndrome / myelodysplastic syndromes / acute myeloid leukaemia / acute chest syndrome / primary myelofibrosis / leukaemia / hereditary angioedema / Maroteaux-Lamy syndrome / C1esterase-inhibitor deficiency / Lennox Gastaut syndrome / cystic fibrosis / hairy cell leukaemia / high-grade resectable non-metastatic osteosarcoma / Pompe disease / Lambert-Eaton myasthenic syndrome / acid α-glucosidase deficiency / chronic iron / Mucopolysaccharidosis VI / disease / secondary acute myeloid leukaemia / patent ductus arteriosus / pulmonary hypertension / Hodgkin lymphoma / symptomatic Sickle Cell Syndrome / N-acetylglutamate synthase primary deficiency / familial lipoprotein lipase deficiency / connective tissue disease / refractory systemic anaplastic large cell lymphoma / /

MedicalTreatment

surgery / oral therapy / blood transfusions / adjunctive therapy / chemotherapy / enzyme replacement therapy / /

Organization

World Health Organization / Takeda Global Research and Development Centre / Europäischen Union / European Medicines Agency / /

Person

Lennox Gastaut / /

Position

Hunter / /

Product

ORFADIN / Orphan-Drug / LYSODREN / INCRELEX / Cystadane / Dexrazoxane / SPRYCEL / Ibuprofen / NEXAVAR / Tasigna / NAGLAZYME / REVLIMID / DACOGEN / EXJADE / dexamethasone / Cladribine / Soliris / PRIALT / Mannitol / REVATIO / KUVAN / MYOZYME / Caffeine citrate / Hydrocortisone / ELAPRASE / Sildenafil citrate / /

Technology

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

URL

http /

SocialTag