573 results
Keyword Amoxicillina ABC Remove keyword
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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 (updated)
Date of designation: 02/02/2010, Positive, Last updated: 13/03/2023vector 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Date of designation: 07/02/2009, Withdrawn, Last updated: 16/10/2014serotype 5 containing the human ABCA4 gene Overview Please note … serotype 5 containing the human ABCA4 gene for the treatment of …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
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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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 … -
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Human medicine European public assessment report (EPAR): Skysona
elivaldogene autotemcel, Adrenoleukodystrophy
Date of authorisation: 16/07/2021,
, Withdrawn, Last updated: 04/04/2022
18 years of age, with an ABCD1 genetic mutation, and for … change (mutation) in the ABCD1 gene. The mutation prevents … -
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Orphan designation: Recombinant human apolipoprotein A-I in a complex with phospholipids for: Treatment of ATP-binding cassette transporter A1 deficiency
Date of designation: 22/08/2014, Positive,cassette transporter A1 (ABCA1) is a protein that regulates … the body. In patients with ABCA1 deficiency, the ABCA1 protein is not produced in … mutation (defect) in their ABCA1 genes, and so cholesterol … -
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Orphan designation: Allogeneic ABCB5-positive limbal stem cells for: Treatment of limbal stem cell deficiency
Date of designation: 14/12/2018, Positive, Last updated: 11/03/2019Allogeneic ABCB5-positive limbal stem cells Overview … KG, Germany, for allogeneic ABCB5-positive limbal stem cells (also known … -
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Orphan designation: Ecothiopate iodide for: Treatment of Stargardt's disease
Date of designation: 24/04/2015, Withdrawn, Last updated: 10/12/2020abnormalities in a gene called ABCA4 . The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: Soraprazan for: Treatment of Stargardt's disease
Date of designation: 13/11/2013, Positive, Last updated: 02/12/2013abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: Emixustat hydrochloride for: Treatment of Stargardt's disease
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt’s disease, ABCR does not work properly. As … -
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Orphan designation: Ramiprilat for: Treatment of Stargardt's disease
Date of designation: 12/03/2013, Positive, Last updated: 17/01/2023abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one (Tinlarebant) for: Treatment of Stargardt's disease
Date of designation: 25/05/2018, Positive, Last updated: 01/12/2022abnormalities in a gene called ABCA4 . The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt's disease, ABCR does not work properly. This … -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Positive, Last updated: 24/05/2022abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … with Stargardt’s disease, ABCR does not work properly. As … -
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Orphan designation: Etidronate disodium for: Treatment of pseudoxanthoma elasticum
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023medicine for the treatment of ABCC6 deficiency in the European … medicine is expected to work The ABCC6 gene is involved in transporting … inherited mutations in the ABCC6 gene, ATP is insufficiently … -
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Human medicine European public assessment report (EPAR): Iscover
clopidogrel, Stroke; Peripheral Vascular Diseases; Atrial Fibrillation; Myocardial Infarction; Acute Coronary Syndrome
Date of authorisation: 14/07/1998, Revision: 49, Authorised, Last updated: 16/02/2023moderate to high-risk TIA (ABCD2 score ≥4) or minor IS … -
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Human medicine European public assessment report (EPAR): Plavix
clopidogrel hydrogen sulfate, Stroke; Peripheral Vascular Diseases; Atrial Fibrillation; Myocardial Infarction; Acute Coronary Syndrome
Date of authorisation: 15/07/1998, Revision: 48, Authorised, Last updated: 15/02/2023moderate to high-risk TIA (ABCD2 score ≥4) or minor IS … -
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Orphan designation: recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 for: Treatment of adenosine triphosphate binding cassette transporter protein subfamily C member 6 deficiency
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021protein subfamily C member 6 (ABCC6) deficiency in the European … expected to work Patients with ABCC6 deficiency have mutations … mutations (changes) in the ABCC6 gene, which controls production … -
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National expert: Martina Cesani, Italian Medicines Agency (updated)
- Declaration of interests - 82.06 KB | PDF
- Curriculum Vitae - 23.46 KB | PDF
5'-half sequence of human ABCA4 gene and the 3'-half sequence … 3'-half sequence of human ABCA4 gene for the treatment of … coding sequences of human ABCA4 fused to inteins; ODD EU/3/19/2208 … -
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News: First gene therapy to treat children with rare inherited neurological disease
CHMP, Last updated: 21/05/2021abnormalities in a gene called ABCD1 which is responsible for … functional copy of the gene ABCD1 for the ALDP protein, so … 18 years of age, with an ABCD1 genetic mutation, and for …