Blood

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Diagnosis, Brain, Bleeding, Disease, Coagulation, Patient, Committee, Knowledge, Regulation, Haemophilia, EMA, IRIS, Joint, Protein, Human, Clinical trial, European, COMP, Spinal cord, Antibody, Factor VIII, Sponsor, European Commission, Immune system, Safety, Blood, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Fabry disease, Brain, Cell, CD34, Disease, Nervous system, Patient, Committee, Knowledge, Tissue, Stroke, Kidney failure, Fats, Regulation, Autotransplantation, EMA, IRIS, Human, Eye, Sugar, European, Skin, COMP, Heart, Sponsor, European Commission, Enzyme replacement therapy, Blood, PPD, Pain, Safety, Marketing, Bone marrow, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Retrieved on: 
Tuesday, April 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, System, Primary, Calcium oxalate, Enzyme, DSM-IV codes, Cell, Disease, Abdominal pain, Patient, Committee, Knowledge, Kidney failure, Organic acid, Regulation, EMA, IRIS, Glycine, Protein, Human, AGXT, Urine, European, COMP, Urinary tract infection, European Commission, Kidney, Safety, Blood, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Community, Eurostat, Civil service commission, Depression, Diagnosis, Psychosis, Cell, Disease, Seizure, Nervous system, Patient, Committee, Knowledge, Light, Regulation, Anxiety, Liver transplantation, EMA, IRIS, AIP, Human, Urine, European, Skin, COMP, Oxygen, Porphyria, European Commission, Safety, Blood, Marketing, Liver, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Diagnosis, Idiopathic pulmonary fibrosis, Disease, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Hydrogen, IPF, European, Collagen, COMP, Oxygen, Urinary tract infection, Sponsor, European Commission, Safety, Blood, Marketing, Medicine, Pharmaceutical industry

Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Orphan designation: 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile Treatment of von Hippel-Lindau disease, 21/08/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Growth, Blood, Cell, Disease, Laser, Patient, Committee, Knowledge, Neoplasm, Regulation, EMA, IRIS, Eye, European, COMP, Pancreas, Spinal cord, Sponsor, European Commission, Von Hippel–Lindau disease, Gland, Safety, VHL, Marketing, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile Treatment of von Hippel-Lindau disease, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile Treatment of von Hippel-Lindau disease, 21/08/2020 Withdrawn

Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Finger, Growth, Face, Blood, Disease, Inflammation, Patient, Committee, Knowledge, Regulation, Systemic scleroderma, EMA, IRIS, Protein, Scleroderma, CB2, European, Collagen, COMP, Connective tissue, Skin, Heart, Sponsor, European Commission, Immune system, The, Safety, Swelling, Marketing, Pharmaceutical industry

Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Key Points: 


Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, European, COMP, Bleeding, Spinal cord, Antibody, Sponsor, European Commission, Immune system, Safety, Factor IX, Blood, Marketing, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Key Points: 


Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Gedeon Richter Selects Trial Interactive for Clinical Trial Management

Retrieved on: 
Monday, March 25, 2024
Biotechnology, Cardiology, Health, Diabetes, Gedeon Richter, CNS, Rheumatology, Trial, Life, Neuropsychiatry, CFR, TMF, Osteoporosis, Blood, Pharmaceutical industry

The Trial Interactive eClinical platform was designed by clinical professionals for clinical professionals.

Key Points: 
  • The Trial Interactive eClinical platform was designed by clinical professionals for clinical professionals.
  • The Trial Interactive platform comprises clinical research-focused products that simplify tasks, oversight, compliance, and audits/inspections.
  • “The Trial Interactive eTMF will help us streamline our TMF operations and maintain inspection readiness.
  • Trial Interactive Division President Michael Smyth commented, “We’re proud to be part of Gedeon Richter’s plans for new studies and look forward to working more closely with their clinical operations teams through our TMF services.”