{"id":229,"date":"2024-05-20T21:23:52","date_gmt":"2024-05-20T19:23:52","guid":{"rendered":"https:\/\/www.dkcommunications.de\/en\/?page_id=229"},"modified":"2024-07-17T21:46:53","modified_gmt":"2024-07-17T19:46:53","slug":"orphan-drugs","status":"publish","type":"page","link":"https:\/\/www.dkcommunications.de\/en\/expertise\/orphan-drugs\/","title":{"rendered":"Orphan Drugs"},"content":{"rendered":"\n

Aegerion<\/strong>
Homozygous familial hypercholesterolemia  (HoFH)<\/p>\n\n\n\n

CSL Behring<\/strong>
Primary and secondary immunodeficiencies(PID, SID); chronic inflammatory demyelinating polyneuropathy (CIDP); alpha 1 antitrypsin deficiency<\/p>\n\n\n\n

EUSA<\/strong>
Neuroblastoma<\/p>\n\n\n\n

Hormosan<\/strong>
Non-dystrophic myotonia (NDM)<\/p>\n\n\n\n

Insmed<\/strong>
Infections with non-tuberculous mycobacteria (NTM)<\/p>\n\n\n\n

Jerini<\/strong>
Hereditary angioedema (HAE)<\/p>\n\n\n\n

Novartis Oncology<\/strong>
Myelofibrosis; pancreatic neuroendocrine tumor (pNET); chronic myeloid leukemia (CML)<\/p>\n\n\n\n

PTC Therapeutics<\/strong>
Duchenne muscular dystrophy with nonsense mutation (nmDMD)<\/p>\n\n\n\n

Sanofi Genzyme<\/strong>
M. Fabry, M. Pompe, M. Gaucher (lysosomal storage diseases); Acquired thrombotic thrombocytopenic purpura (aTTP)<\/p>\n","protected":false},"excerpt":{"rendered":"

AegerionHomozygous familial hypercholesterolemia  (HoFH) CSL BehringPrimary and secondary immunodeficiencies(PID, SID); chronic inflammatory demyelinating polyneuropathy (CIDP); alpha 1 antitrypsin deficiency EUSANeuroblastoma HormosanNon-dystrophic myotonia (NDM) InsmedInfections with non-tuberculous mycobacteria (NTM) JeriniHereditary angioedema (HAE) Novartis OncologyMyelofibrosis; pancreatic neuroendocrine tumor (pNET); chronic myeloid leukemia (CML) PTC TherapeuticsDuchenne muscular dystrophy with nonsense mutation (nmDMD) Sanofi GenzymeM. Fabry, M. Pompe, M. […]<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":224,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"page-templates\/page-sidebar-none.php","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-229","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nOrphan Drugs - dk Life Science Communications<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.dkcommunications.de\/en\/expertise\/orphan-drugs\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Orphan Drugs - dk Life Science Communications\" \/>\n<meta property=\"og:description\" content=\"AegerionHomozygous familial hypercholesterolemia  (HoFH) CSL BehringPrimary and secondary immunodeficiencies(PID, SID); chronic inflammatory demyelinating polyneuropathy (CIDP); alpha 1 antitrypsin deficiency EUSANeuroblastoma HormosanNon-dystrophic myotonia (NDM) InsmedInfections with non-tuberculous mycobacteria (NTM) JeriniHereditary angioedema (HAE) Novartis OncologyMyelofibrosis; pancreatic neuroendocrine tumor (pNET); chronic myeloid leukemia (CML) PTC TherapeuticsDuchenne muscular dystrophy with nonsense mutation (nmDMD) Sanofi GenzymeM. Fabry, M. Pompe, M. 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