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genetic disease
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Editing Tool Works as a 'guided Missile' to Correct Errors in The Genetic Code
Feb 26, 2014Work uncovers secret of how genome-editing tool works as a 'guided missile' to correct errors in the genetic code Researchers from the Broad Institute and MIT have teamed up with colleagues from the University of Tokyo to form the first ...
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OvaScience and Intrexon Corporation Have Announced an Agreement
Dec 23, 2013OvaScience, a life sciences company, and Intrexon Corporation have announced an agreement to access Intrexon’s portfolio of technologies to accelerate the development of OvaScience’s OvaTure technology platform Separately, ...
Tags: OvaScience, Infertility Treatments
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A Gene Therapy Company Focused on Developing Novel Treatments for Rare Diseases
Nov 4, 2013Fidelity Biosciences, a subsidiary of Fidelity Investments, and US-based Regenx Biosciences have announced the formation of Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Dimension ...
Tags: Biosciences, Health, Medicine
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Japan Approvals The Use of Soliris (Eculizumab) Drug to Treat aHUS
Sep 17, 2013Biotechnology firm Alexion Pharmaceuticals has secured approval from the Ministry of Health, Labour and Welfare (MHLW) in Japan for the use of its Soliris (eculizumab) drug to treat pediatric and adult patients with atypical hemolytic ...
Tags: Ahus Treatment, Soliris Drug
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Plexcera Therapeutics Has Received Approval From MSIP
Sep 17, 2013Plexcera Therapeutics has received approval from Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine, to develop recombinant human acid ceramidase (rhAC) for treatment of diseases caused by genetic or ...
Tags: Plexcera, Cystic Fibrosis
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Sangamo BioSciences Has Received a $6.4m Strategic Partnership Grant From The CIRM
May 27, 2013Sangamo BioSciences has received a $6.4m strategic partnership grant from the California Institute for Regenerative Medicine (CIRM) for the development of ZFP Therapeutic for Beta-thalassemia. The application of its zinc finger nuclease ...
Tags: Sangamo, ZFG Therapeutic
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Aegerion Has Received US FDA Approval to Be Used as an Adjunct to a Low-Fat Diet
Dec 28, 2012Aegerion Pharmaceuticals' Juxtapid (lomitapide) capsules has received US FDA approval to be used as an adjunct to a low-fat diet and other lipid-lowering treatments. Juxtapid, which is a supplement to fat reduction treatments also ...
Tags: Juxtapid, US, FDA, capsules, treatments
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ISIS Pharmaceuticals Has Obtained The US FDA Fast Track Designation for ISIS-TTR
Dec 17, 2012Isis Pharmaceuticals has obtained the US FDA fast track designation for ISIS-TTR to treat familial amyloid polyneuropathy (FAP). The antisense drug ISIS-TTR, a part of the Isis-GSK strategic RNA therapeutic alliance, is being developed ...
Tags: amyloid polyneuropathy candidate, Pharmaceuticals, genetic disease, drug
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A Sequencing Protocol Using a Rapid-Throughput Platform Could Help Diagnose Ill Infants
Dec 11, 2012Illumina system used in faster genome sequencing 08 Oct 2012 A sequencing protocol using a rapid-throughput platform could help diagnose critically ill infants. Faster sequencing Investigators at Kansas City's Children's Mercy ...
Tags: Illumina system, Science Translational Medicine, test, health
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Protalix BioTherapeutics Signed Agreement to Advance Gaucher Disease Drug Trials
Dec 10, 2012Israel-based Protalix BioTherapeutics has signed an agreement with Pfizer to continue managing the development activities of Gaucher disease drug Elelyso. According to the clinical development agreement, Protalix will continue to manage ...
Tags: Protalix, Pfizer, Gaucher disease drug
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European Commission Has Approved Vertex Pharmaceuticals's Kalydeco
Jul 30, 2012The European Commission has approved Vertex Pharmaceuticals' Kalydeco (ivacaftor) to treat the underlying cause of cystic fibrosis in people with a specific genetic mutation (G551D). The approval is based on positive findings from two ...
Tags: european approval, kalydeco, cystic fibrosis
