Acute Intermittent Porphyria News and Research

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New acute porphyria treatment shows promising results in clinical study

Acute porphyria is a group of uncommon diseases that can cause severe, potentially life-threatening attacks of abdominal pain, nausea, vomiting and paralysis. Liver transplantation is currently the only effective treatment available for the most seriously afflicted patients.

7 Feb 2019

New pre-clinical research shows utility of mRNA platform to treat rare metabolic disorders

An mRNA platform that can express therapeutic levels of protein in liver tissue could be used to treat patients with rare metabolic disorders.

9 Oct 2018

Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today positive topline results from the interim analysis of the ENVISION Phase 3 Study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria.

27 Sep 2018

Wrecked and retching: Obscure vomiting illness linked to long-term pot use

For 17 years, Chalfonte LeNee Queen suffered periodic episodes of violent retching and abdominal pain that would knock her off her feet for days, sometimes leaving her writhing on the floor in pain.

2 Jan 2018

New gene therapy for Acute Intermittent Porphyria

Angeles suffers from a severe and rare genetic disease called Acute Intermittent Porphyria (AIP). This means, one of her genes restrains her liver to produce a specific protein needed for the metabolism of the blood. During an AIP attack Angeles usually feels intense abdominal pain, and her mental status changes. Women live at an increased risk to trigger the symptoms, because porphyria is related to the menstrual cycle.

2 Jul 2014

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

uniQure commences acute intermittent porphyria Phase I clinical trial

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.

11 Dec 2012

uniQure receives positive recommendation from EMA CHMP for Glybera

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

21 Jul 2012

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2011.

25 Aug 2011

AMT 2010 total net income increases to EUR 1.4 million

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010.

16 Feb 2011

Digna Biotech, CIMA receive EU grant for development of gene therapy product for Acute Intermittent Porphyria

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).

4 Feb 2011

EU grants 3.3 million to AMP for gene therapy development against AIP

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Union 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.

31 Jan 2011

AMT announces full year 2009 results; reports modest decrease in operating expenses

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009.

24 Feb 2010

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2009.

18 Nov 2009

Treatment for acute intermittent porphyria

The Center for Applied Medical Research (CIMA) from the University of Navarra and the Amsterdam Molecular Therapeutics BV (AMT) have demonstrated the pre-clinical effectiveness of a treatment against acute intermittent porphyria.

2 Apr 2007