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42 results
Orphan designation – intended use
Treatment of amyotrophic lateral sclerosis Remove Treatment of amyotrophic lateral sclerosis filter
Orphan designation status
Positive Remove Positive filter
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2014, Positive, Last updated: 15/12/2022 -
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Orphan designation: Human culture expanded autologous mesenchymal stromal cells for: Treatment of amyotrophic lateral sclerosis
Date of designation: 24/04/2019, Positive, Last updated: 03/05/2022 -
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Orphan designation: Pridopidine hydrochloride for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/07/2021, Positive, Last updated: 17/02/2022 -
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Orphan designation: Sodium phenylbutyrate, Tauroursodeoxycholic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 04/06/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: Enoxacin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/03/2015, Positive, Last updated: 02/09/2021 -
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Orphan designation: (R)-troloxamide quinone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 12/10/2017, Positive, Last updated: 03/12/2019 -
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Orphan designation: Ibudilast for: Treatment of amyotrophic lateral sclerosis
Date of designation: 12/12/2016, Positive, Last updated: 16/10/2019 -
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Orphan designation: S[+] apomorphine for: Treatment of amyotrophic lateral sclerosis
Date of designation: 09/02/2012, Positive, Last updated: 03/10/2019 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/06/2015, Positive, Last updated: 21/05/2019 -
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Orphan designation: Smilagenin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 27/09/2011, Positive, Last updated: 22/02/2019 -
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Orphan designation: H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide) for: Treatment of amyotrophic lateral sclerosis
Date of designation: 30/05/2016, Positive, Last updated: 18/12/2018 -
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Orphan designation: Ambroxol hydrochloride for: Treatment of amyotrophic lateral sclerosis
Date of designation: 25/05/2018, Positive, Last updated: 24/07/2018 -
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Orphan designation: adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1 for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/04/2018, Positive, Last updated: 17/05/2018 -
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Orphan designation: Sodium chlorite for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/06/2013, Positive, Last updated: 03/01/2018 -
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Orphan designation: Recombinant human antibody directed against misfolded human superoxide dismutase 1 for: Treatment of amyotrophic lateral sclerosis
Date of designation: 17/07/2017, Positive, Last updated: 14/08/2017 -
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Orphan designation: 6-Ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one (tirasemtiv) for: Treatment of amyotrophic lateral sclerosis
Date of designation: 05/03/2012, Positive, Last updated: 17/07/2017 -
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Orphan designation: Tauroursodeoxycholic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 27/02/2017, Positive, Last updated: 31/03/2017 -
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Orphan designation: Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: Masitinib mesilate for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: Recombinant human cerebral dopamine neurotrophic factor for: Treatment of amyotrophic lateral sclerosis
Date of designation: 28/04/2016, Positive, Last updated: 30/05/2016 -
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Orphan designation: Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate for: Treatment of amyotrophic lateral sclerosis
Date of designation: 28/07/2015, Positive, Last updated: 18/08/2015 -
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Orphan designation: filgrastim for: Treatment of amyotrophic lateral sclerosis
Date of designation: 01/04/2008, Positive, Last updated: 26/03/2015 -
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Orphan designation: Recombinant human vascular endothelial growth factor for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/01/2010, Positive, Last updated: 12/05/2014 -
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Orphan designation: Autologous bone-marrow-derived mesenchymal stromal cells secreting neurotrophic factors for: Treatment of amyotrophic lateral sclerosis
Date of designation: 17/07/2013, Positive, Last updated: 16/08/2013 -
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Orphan designation: Allogeneic motor neuron progenitor cells derived from human embryonic stem cells for: Treatment of amyotrophic lateral sclerosis
Date of designation: 17/07/2013, Positive, Last updated: 16/08/2013