1521 results
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Orphan designation: Mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene for: Treatment of Stargardt's disease
Date of designation: 04/07/2014, Positive, Last updated: 07/11/20225'-half sequence of human ABCA4 gene and the 3'-half sequence … 3'-half sequence of human ABCA4 gene Overview On 4 July 2014 … 5'-half sequence of human ABCA4 gene and the 3'-half sequence … -
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Orphan designation: Lentiviral vector containing the human ABCA4 gene for: Treatment of Stargardt's disease
Date of designation: 02/02/2010, Positive, Last updated: 08/02/2016vector containing the human ABCA4 gene Overview On 2 February … vector containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The … -
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Opinion/decision on a Paediatric investigation plan (PIP): Lentiviral vector containing the human ABCA4 gene
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Ophthalmology
PIP number: EMEA-002407-PIP01-18, Route(s) of administration: Intraocular use, Pharmaceutical form(s): Solution for injection
Decision date: 14/06/2019, Last updated: 07/11/2019, Compliance check: Xvector containing the human ABCA4 gene OphthalmologyP/0213/2019EMEA-002407-PIP01-18 … vector containing the human ABCA4 gene (SAR422459) (EMEA-002407-PIP01-18 … vector containing the human ABCA4 gene … -
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Orphan designation: Adeno-associated viral vector serotype 5 containing the human ABCA4 gene for: Treatment of Stargardt's disease
Date of designation: 07/02/2009, Withdrawn, Last updated: 16/10/2014serotype 5 containing the human ABCA4 gene Overview Please note … serotype 5 containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The … -
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Orphan designation: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells for: Treatment of epidermolysis bullosa
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019Allogeneic skin-derived ABCB5-positive mesenchymal stem cells Overview … for allogeneic skin-derived ABCB5-positive mesenchymal stem cells (also … of allogeneic skin-derived ABCB5-positive mesenchymal stem cells have … -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human ABCD1 gene for: Treatment of adrenoleukodystrophy (new)
Date of designation: 11/11/2022, Positive, Last updated: 25/01/2023serotype 9 containing the human ABCD1 gene Overview This medicine … serotype 9 containing the human ABCD1 gene … -
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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
Date of designation: 06/06/2012, Withdrawn, Last updated: 04/04/2022Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel … Lenti-D encoding the human ABCD1 cDNA was authorised in the … Lenti-D encoding the human ABCD1 cDNA for the treatment of … -
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Referral: Serotonin-specific reuptake inhibitors and serotonin-noradrenaline reuptake inhibitors
atomoxetine, citalopram, escitalopram, fluoxetine, fluvoxamine, mianserine, milnacipran, mirtazapine, paroxetine, reboxetine, sertraline, venlafaxine, Article 31 referrals
Status: European Commission final decision, opinion/position date: 21/04/2005, EC decision date: 19/08/2005, Last updated: 03/11/2005substances: atomoxetine, citalopram, duloxetine, escitalopram … SSRIs / SNRIs: atomoxetine, citalopram, duloxetine, escitalopram … the product information of citalopram, duloxetine 1 , escitalopram … -
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Orphan designation: Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins for: Treatment of Stargardt's disease
Date of designation: 17/10/2019, Positive, Last updated: 07/11/2022coding sequences of human ABCA4 fused to inteins Overview … coding sequences of human ABCA4 fused to inteins for the … abnormalities in a gene called ABCA4. The … -
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Opinion/decision on a Paediatric investigation plan (PIP): Allogeneic skin-derived ATP-binding cassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem cells (allo-APZ2-EB)
Decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Therapeutic area: Dermatology
PIP number: EMEA-002875-PIP01-20, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Suspension for injection
Decision date: 09/07/2021, Last updated: 19/07/2022, Compliance check: Xcassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem … cassette, sub-family B, member 5 (ABCB5)-positive mesenchymal stem … sub-family B, member 5 (ABCB5)-positive mesenchymal stem … -
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Opinion/decision on a Paediatric investigation plan (PIP): autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ATP-binding cassette, sub-family D (ALD), member 1 (ABCD1) from cDNA
Decision type: PM: decision on the application for modification of an agreed PIP
Therapeutic area: Neurology
PIP number: EMEA-001244-PIP01-11-M02, Route(s) of administration: Intravenous use, Pharmaceutical form(s): Dispersion for infusion
Decision date: 12/09/2018, Last updated: 16/09/2021, Compliance check: V, 04/09/2020sub-family D (ALD), member 1 (ABCD1) from cDNA NeurologyP/0290/2018EMEA-001244-PIP01-11-M02 … sub-family D (ALD), member 1 (ABCD1) from cDNA …