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13 results
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Orphan designation: Rintatolimod for: Treatment of pancreatic cancer
Date of designation: 19/02/2022, Positive, Last updated: 22/02/2022 -
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Orphan designation: Valemetostat tosilate for: Treatment of peripheral T-cell lymphoma
Date of designation: 24/02/2022, Positive, Last updated: 28/04/2022 -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of designation: 24/02/2022, Positive, Last updated: 28/04/2022 -
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Orphan designation: Vatiquinone for: Treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes
Date of designation: 24/02/2022, Positive, Last updated: 28/04/2022 -
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Orphan designation: Bovactant for: Treatment of respiratory distress syndrome
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: Gadolinium-chelated polysiloxane nanoparticles for: Treatment of pancreatic cancer
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: [Ala1,3,12,Gln10,Arg11,Trp14]PTH(1-14)/[Ala18,22, Lys26]PTHrP(15-36)COOH for: Treatment of hypoparathyroidism
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: aldesleukin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: Epcoritamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 24/02/2022, Positive, Last updated: 13/05/2022 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of Ehlers-Danlos syndrome
Date of designation: 24/02/2022, Positive, Last updated: 13/05/2022 -
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Orphan designation: 18‐mer antisense oligonucleotide complementary to SCN1A mRNA, sodium salt for: Treatment of Dravet syndrome
Date of designation: 24/02/2022, Positive, Last updated: 08/09/2022 -
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Orphan designation: Autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR gene for: Treatment of pyruvate kinase deficiency
Date of designation: 24/02/2022, Positive, Last updated: 11/11/2022 -
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Orphan designation: Cannabidiol for: Treatment of fragile X syndrome (FXS) (updated)
Date of designation: 25/02/2022, Positive, Last updated: 07/03/2023