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17 results
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Orphan designation: begelomab for: Treatment of dermatomyositis
Date of designation: 20/05/2021, Positive, Last updated: 16/03/2022 -
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Orphan designation: Macitentan for: Treatment of functional single ventricle congenital heart disease
Date of designation: 20/05/2021, Positive, Last updated: 16/03/2022 -
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Orphan designation: Eflornithine for: Treatment of neuroblastoma
Date of designation: 20/05/2021, Positive, Last updated: 17/03/2022 -
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Orphan designation: Elamipretide for: Treatment of Barth syndrome
Date of designation: 20/05/2021, Positive, Last updated: 17/03/2022 -
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Orphan designation: Human IgG1 monoclonal antibody against alpha-synuclein for: Treatment of multiple system atrophy
Date of designation: 20/05/2021, Positive, Last updated: 17/03/2022 -
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Orphan designation: 2-[4-[3-(methylamino)-1-phenylpropoxy]phenyl]ethanol hydrochloride for: Treatment of fragile X syndrome
Date of designation: 20/05/2021, Positive, Last updated: 17/03/2022 -
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Orphan designation: Adeno-associated viral vector serotype 2.5T encoding the human cystic fibrosis transmembrane conductance regulator with a partial deletion in the R domain for: Treatment of cystic fibrosis
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: 6-Amino-5-chloro-N-((1R)-1-(5-(((5-chloro-4-(trifluoromethyl)-2-pyridinyl)amino)carbonyl)-2-thiazolyl)ethyl)-4-pyrimidinecarboxamide for: Treatment of glioma
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one for: Treatment of hyperphenylalaninemia
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: Trehalose for: Treatment of amyotrophic lateral sclerosis
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: Synthetic 2′-O-(2-methoxyethyl)-modified antisense oligonucleotide linked to a triantennary cluster of N-acetyl galactosamine sugars targeting transmembrane protease, serine 6 mRNA for: Treatment of beta thalassaemia intermedia and major
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: zanubrutinib for: Treatment of marginal zone lymphoma
Date of designation: 20/05/2021, Withdrawn, Last updated: 05/01/2023 -
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Orphan designation: melatonin for: Treatment of retinitis pigmentosa
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene for: Treatment of X-linked severe combined immunodeficiency
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: Adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene for: Treatment of Leber's congenital amaurosis
Date of designation: 20/05/2021, Positive, Last updated: 18/03/2022 -
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Orphan designation: Adeno-associated virus serotype 9 expressing the cDNA for human MECP2 for: Treatment of Rett syndrome
Date of designation: 20/05/2021, Withdrawn, Last updated: 30/03/2022 -
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Orphan designation: Dantrolene sodium, hemiheptahydrate for: Treatment of malignant hyperthermia
Date of designation: 20/05/2021, Positive, Last updated: 24/03/2022