Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2012-04-04 | interferon gamma | Prof. Roberto Testi (Italy) | Friedreich’s ataxia | |
2012-04-04 | human haptoglobin | Bio Products Laboratory (UK) | sickle cell disease | |
2012-04-04 | cysteamine | NovaBiotics (UK) | cystic fibrosis |
Granting of the orphan status in the US |
2012-04-04 | adeno-associated viral vector serotype 8 containing the human AIPL1 gene | Fondazione Telethon (Italy) | Leber’s congenital amaurosis type 4 | Granting of the orphan status in the EU |
2012-04-04 | 4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol | Celgene Europe (UK), subsidiary of Celgene (USA) | idiopathic pulmonary fibrosis |
Granting of the orphan status in the EU |
2012-04-04 | mono-pegylated Interferon alpha 2b | AOP Orphan Pharmaceuticals (Austria) | polycythaemia vera |
Granting of a patent |
2012-04-04 | alpha-tocotrienol quinone | Edison Europe Holding (The Netherlands) | Leigh syndrome | |
2012-04-04 | plerixafor | Genzyme (USA - MA), a Sanofi company (France) | adjunctive treatment to cytotoxic therapy in acute myeloid leukaemia (AML) |
Granting of the orphan status in the EU |
2012-04-04 | recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D | Syntaxin (UK) | acromegaly |
Granting of the orphan status in the EU |
2012-04-04 | ornithine phenylacetate | Dr Ulrich Granzer (Germany) | acute liver failure | |
2012-04-04 | liposomal combination of cytarabine and daunorubicin | Celator (UK) | acute myeloid leukaemia |
|
2012-04-04 | lipopolysaccharide of Ochrobactrum intermedium | Diomune (Spain) | prevention of sepsis in at-risk premature infants of less than or equal to 32 weeks of gestational age | |
2012-04-04 | recombinant homodimer of the human annexin V | Astellas Pharma Europe (The Netherlands), subsidiary of Astellas Pharma (Japan) | prevention of ischaemia/reperfusion injury associated with solid organ transplantation | |
2012-04-04 | chimeric locked nucleic acid-deoxynucleoside phosphorothioate-linked oligonucleotide directed against microRNA-451 | Miragen Therapeutics Europe (UK) | polycythaemia vera | |
2012-01-11 | sodium phenylbutyrate | GMP Orphan (France) | 5q spinal muscular atrophy |
Granting of the orphan status in the US |
2012-04-04 | N,N\'-bis(2-mercaptoethyl)isophthalamide | CTI Science (Ireland) | mercury toxicity | |
2012-04-04 | human monoclonal antibody against Fas ligand | PinCell (Italy) | pemphigus | |
2012-04-05 | autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene | Généthon (France) | X-linked chronic granulomatous disease | Granting of the orphan status in the EU |
2012-04-05 | sodium phenylbutyrate | Lucane Pharma (France) | ornithine transcarbamylase deficiency | |
2012-04-05 | sodium phenylbutyrate | Lucane Pharma (France) | citrullinaemia type 1 |
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