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1775 results
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) for: Treatment of adrenoleukodystrophy (updated)
Date of designation: 06/06/2012, Positive, Last updated: 27/07/2021 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency (updated)
Date of designation: 14/07/2016, Positive, Last updated: 22/07/2021 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency (updated)
Date of designation: 19/11/2018, Positive, Last updated: 22/07/2021 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of systemic sclerosis (updated)
Date of designation: 15/05/2009, Positive, Last updated: 16/07/2021 -
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Orphan designation: Ralinepag for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 11/01/2019, Positive, Last updated: 16/07/2021 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension (updated)
Date of designation: 26/08/2005, Positive, Last updated: 16/07/2021 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension (updated)
Date of designation: 14/04/2004, Positive, Last updated: 16/07/2021 -
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Orphan designation: (R-S) baclofen, naltrexone hydrochloride, D-sorbitol for: Treatment of Charcot-Marie-Tooth disease type 1A (updated)
Date of designation: 26/03/2014, Positive, Last updated: 16/07/2021 -
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Orphan designation: Adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec) for: Treatment of glioma (updated)
Date of designation: 06/06/2012, Positive, Last updated: 16/07/2021 -
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Orphan designation: Ofranergene obadenovec for: Treatment of ovarian cancer (updated)
Date of designation: 16/10/2017, Positive, Last updated: 16/07/2021 -
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Orphan designation: Human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab) for: Treatment of pneumonia caused by Staphylococcus aureus (updated)
Date of designation: 05/03/2012, Positive, Last updated: 13/07/2021 -
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Orphan designation: gallium citrate for: Treatment of cystic fibrosis (updated)
Date of designation: 21/08/2019, Positive, Last updated: 13/07/2021 -
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Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa serotype O11 (Panobacumab) for: Treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa (updated)
Date of designation: 29/06/2006, Positive, Last updated: 13/07/2021 -
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Orphan designation: Adenovirus-associated viral vector serotype 5 containing the human RPGR gene for: Treatment of retinitis pigmentosa (updated)
Date of designation: 29/08/2016, Positive, Last updated: 12/07/2021 -
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Orphan designation: Adenovirus associated viral vector serotype 5 containing the human RPE65 gene for: Treatment of Leber's congenital amaurosis (updated)
Date of designation: 11/11/2015, Positive, Last updated: 12/07/2021 -
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Orphan designation: Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene for: Treatment of Leber's congenital amaurosis (updated)
Date of designation: 12/12/2017, Positive, Last updated: 12/07/2021 -
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Orphan designation: Adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene for: Treatment of achromatopsia (updated)
Date of designation: 31/07/2018, Positive, Last updated: 12/07/2021 -
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Orphan designation: Adeno-associated virus serotype 5 containing the human RDH12 gene for: Treatment of RDH12 mutation associated retinal dystrophy (updated)
Date of designation: 19/10/2020, Positive, Last updated: 12/07/2021 -
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Orphan designation: budesonide for: Treatment of primary IgA nephropathy (updated)
Date of designation: 18/11/2016, Positive, Last updated: 12/07/2021 -
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Orphan designation: dimethyl fumarate for: Adjunctive treatment in haematopoietic cell transplantation (updated)
Date of designation: 21/03/2018, Positive, Last updated: 08/07/2021 -
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Orphan designation: Mixture of seven synthetic fragments consisting of p21 RAS peptides for: Treatment of pancreatic cancer (updated)
Date of designation: 05/08/2011, Positive, Last updated: 08/07/2021 -
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity (updated)
Date of designation: 03/02/2003, Positive, Last updated: 06/07/2021 -
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Orphan designation: Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec) for: Treatment of haemophilia B (updated)
Date of designation: 21/03/2018, Positive, Last updated: 01/07/2021 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome (updated)
Date of designation: 25/05/2018, Positive, Last updated: 01/07/2021 -
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Orphan designation: "Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus (bulevirtide) for: Treatment of hepatitis delta virus infection (updated)
Date of designation: 19/06/2015, Positive, Last updated: 30/06/2021