440 results
Keyword Bromazepam 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 first decision: 04/07/2014, Positive, Last updated: 26/03/20155'-half sequence of human ABCA4 gene and the 3'-half sequence … 3'-half sequence of human ABCA4 gene for the treatment of … 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 first decision: 02/02/2010, Positive, Last updated: 08/02/2016vector containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR, which controls the movement … -
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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 Therapeutic area Ophthalmology … 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Date of first decision: 07/02/2009, Withdrawn, Last updated: 16/10/2014serotype 5 containing the human ABCA4 gene for the treatment of … abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … -
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Orphan designation: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019for allogeneic skin-derived ABCB5-positive mesenchymal stem cells (also … of allogeneic skin-derived ABCB5-positive mesenchymal stem cells have … Allogeneic skin-derived ABCB5-positive mesenchymal stem cells … -
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA for: Treatment of adrenoleukodystrophy
Date of first decision: 06/06/2012, Positive, Last updated: 04/03/2019Lenti-D encoding the human ABCD1 cDNA for the treatment of … abnormalities in a gene called ABCD1 which is responsible for … functional copy of the gene ABCD1 for the ALDP protein, so … -
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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 first decision: 17/10/2019, Positive, Last updated: 22/01/2020coding sequences of human ABCA4 fused to inteins for the … abnormalities in a gene called ABCA4. The ABCA4 gene is responsible for the … production of a protein called ABCR that regulates the transport … -
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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: 29/01/2019, Compliance check: Xsub-family D (ALD), member 1 (ABCD1) from cDNA Therapeutic area … sub-family D (ALD), member 1 (ABCD1) from cDNA … -
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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 first decision: 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: 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 for: Treatment of Stargardt's disease
Date of first decision: 25/05/2018, Positive, Last updated: 23/07/2018abnormalities 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 first decision: 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: Allogeneic ABCB5-positive limbal stem cells for: Treatment of limbal stem cell deficiency
Date of first decision: 14/12/2018, Positive, Last updated: 11/03/2019KG, Germany, for allogeneic ABCB5-positive limbal stem cells (also known … -
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Orphan designation: Emixustat hydrochloride for: Treatment of Stargardt's disease
Date of first decision: 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: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of first decision: 24/04/2019, Positive, Last updated: 14/06/2019abnormalities 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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Press release: European Medicines Agency finalises review of medicines concerned by Roche pharmacovigilance inspection
Last updated: 19/11/2013allopurinol, benzerapide/levodopa, bromazepam, calcitriol, carvedilol, ceftriaxone … allopurinol, benzerapide/levodopa, bromazepam, calcitriol, carvedilol, ceftriaxone … -
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Orphan designation: Ecothiopate iodide for: Treatment of Stargardt's disease
Date of first decision: 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: Ramiprilat for: Treatment of Stargardt's disease
Date of first decision: 12/03/2013, Positive, Last updated: 05/04/2013abnormalities 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: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride for: Treatment of adrenoleukodystrophy
Date of first decision: 18/11/2016, Positive, Last updated: 13/12/2016abnormalities in a gene called ABCD1 which is responsible for … abnormalities in a gene called ABCD1 which is responsible for … -
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Orphan designation: Temsirolimus for: Treatment of adrenoleukodystrophy
Date of first decision: 30/05/2016, Positive, Last updated: 24/06/2016abnormalities in a gene called ABCD1 which is responsible for … abnormalities in a gene called ABCD1 which is responsible for … -
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Orphan designation: pioglitazone for: Treatment of adrenoleukodystrophy
Date of first decision: 19/02/2014, Positive, Last updated: 02/04/2014abnormalities in a gene called ABCD1 which is responsible for … abnormalities in a gene called ABCD1 which is responsible for … -
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Orphan designation: Human embryonic stem-cell-derived retinal pigment epithelial cells for: Treatment of Stargardt's disease
Date of first decision: 21/06/2011, Positive, Last updated: 12/07/2011disease lack a protein called ABCR, which controls the movement … -
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Orphan designation: dimethyl fumarate for: Treatment of adrenoleukodystrophy
Date of first decision: 09/01/2020, Positive, Last updated: 14/05/2020ABCD1 … -
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Human medicine European public assessment report (EPAR): Plavix (updated)
clopidogrel hydrogen sulfate, Stroke, Peripheral Vascular Diseases, Atrial Fibrillation, Myocardial Infarction, Acute Coronary Syndrome
Date of authorisation: 15/07/1998, Revision: 43, Authorised, Last updated: 12/02/2021moderate to high-risk TIA (ABCD2 score ≥4) or minor IS … -
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Human medicine European public assessment report (EPAR): Iscover (updated)
clopidogrel, Stroke, Peripheral Vascular Diseases, Atrial Fibrillation, Myocardial Infarction, Acute Coronary Syndrome
Date of authorisation: 14/07/1998, Revision: 44, Authorised, Last updated: 12/02/2021moderate to high-risk TIA (ABCD2 score ≥4) or minor IS … -
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National expert: Balázs Sarkadi, National Institute of Pharmacy and Nutrition (updated)
- Declaration of interests - 81.53 KB | PDF
- Curriculum Vitae - 235.79 KB | PDF
by overexpression of the ABCG2 multidrug transporter in … parallel measurements of the ABCG2, ABCB1 and ABCC1 multidrug transporter functions PLOS … http://www.google.com/patents/US8129197 ?ABC transporter assay" Balazs …