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Last updated on April 17, 2014 at 17:30 EDT

Latest porphyrias Stories

2012-12-11 04:21:28

AMSTERDAM, December 11, 2012 /PRNewswire/ -- uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and...

2012-07-05 02:23:53

BLAINVILLE, QC, July 5, 2012 /PRNewswire/ - Medunik Canada, is an orphan drug company dedicated to helping Canadians with rare diseases access therapies currently unavailable in Canada. Medunik Canada is proud to announce that it has signed an exclusive distribution agreement with Orphan Europe Recordati Group. Under this agreement, Medunik Canada receives the exclusive Canadian rights to market and distribute four important therapies in the following medical conditions: (1) acute...

2011-02-03 08:59:00

MADRID, February 3, 2011 /PRNewswire/ -- Digna Biotech and the Centro de Investigacion Medica Aplicada (CIMA) de la Universidad de Navarra announced today that the European Union (EU) has finalized a EUR 3.3 million grant to AIPGENE consortium. Digna Biotech and CIMA will receive, as members of the consortium, EUR 0.5 and EUR 0.8 million respectively for the clinical development of a gene therapy product for Acute Intermittent Porphyria (AIP). The AIPGENE consortium is led by CIMA...

2011-01-30 23:59:00

AMSTERDAM, The Netherlands, January 31, 2011 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Union (EU) has finalized a EUR 3.3 million grant to the AIPGENE consortium, of which AMT is a member, for the development of a gene therapy product for Acute Intermittent Porphyria (AIP). AIP is a severe and progressive disease caused by the inability of the body to produce the heme protein,...

2009-05-28 00:00:00

AMSTERDAM, May 28 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP). Orphan Drug Designation for AIP entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-021 if this product candidate is the first...

2005-10-07 13:48:34

HOUSTON "“ (Oct. 7, 2005) -- A research team led by scientists at The University of Texas Medical School at Houston has determined the structure of an enzyme that when defective causes an inherited disease that afflicts sufferers with severe abdominal pain, psychiatric symptoms, skin fragility, and light sensitivity. "Unless prompt and appropriate treatment is given, hereditary coproporphyria can very quickly turn into a life-threatening medical emergency," said C. S. Raman, Ph.D.,...