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12 results
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Orphan designation: Rezafungin acetate for: Treatment of invasive candidiasis
Date of designation: 06/01/2021, Positive, Last updated: 30/06/2022 -
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Orphan designation: sodium oxybate, calcium oxybate, magnesium oxybate, potassium oxybate for: Treatment of idiopathic hypersomnia
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: setanaxib for: Treatment of primary biliary cholangitis
Date of designation: 06/01/2021, Positive, Last updated: 30/08/2022 -
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Orphan designation: Human laminin-111, recombinant for: Treatment of congenital muscular dystrophy
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Cyclo-L-glycyl-L-2-allylproline for: Treatment of Angelman syndrome
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Rilonacept for: Treatment of idiopathic pericarditis
Date of designation: 06/01/2021, Positive, Last updated: 30/01/2023 -
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Orphan designation: idursulfase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 06/01/2021, Withdrawn, Last updated: 27/01/2023 -
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Orphan designation: Humanised IgG1K monoclonal antibody against interferon beta for: Treatment of dermatomyositis
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Cyclo-L-glycyl-L-2-allylproline for: Treatment of Pitt-Hopkins syndrome
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Cyclo-L-glycyl-L-2-allylproline for: Treatment of Phelan-McDermid syndrome
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Celecoxib, Ciprofloxacin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021 -
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Orphan designation: Alpha galactosidase A for: Treatment of Fabry disease
Date of designation: 06/01/2021, Positive, Last updated: 18/05/2021