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Mashup Score: 29
Abstract. The US Food and Drug Administration (FDA)’s authorization of etranacogene dezaparvovec (Hemgenix) is a significant milestone, constituting not on
Source: ashpublications.orgCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 7
After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have broken new boundaries in pricing, too.
Source: www.fiercepharma.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 9CSL Behring Signs First Commercial Agreement in Austria to Fund Haemophilia B Gene Therapy HEMGENIX® (1) - 2 month(s) ago
Global biotechnology leader CSL Behring (ASX: CSL) today announced that a commercial contract has been signed to fund HEMGENIX® (etranacogene dezaparvovec) in Austria, with the option to access a dedicated fund, established by the provinces for specialised medicines. This is the first commercial …
Source: www.pharmiweb.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 17Breaking ground in haemophilia B gene therapy: insights from the HOPE-B trial and beyond - 2 month(s) ago
There are more references available in the full text version of this
Source: www.sciencedirect.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 22
Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophyla…
Source: www.sciencedirect.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 0
Pricing: Free for Members and Non-MembersThis activity is supported by an educational grant from BioMarin Pharmaceutical Inc.Course DescriptionDiagnosis of rare diseases affecting the blood are increasing in incidence due to sophistication in diagnostics and molecular genetic discoveries and are particularly concerning because of the life-threatening complications. Hemophilia describes a duo of…
Source: amcplearn.orgCategories: General Medicine News, PayerTweet
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Mashup Score: 20Center for Inherited Blood Disorders Administers Country's First Gene Therapy Infusion to Treat Hemophilia A - 3 month(s) ago
Today, the Center for Inherited Blood Disorders (CIBD) announced the successful administration of the first gene therapy infusion in the country to tr
Source: www.businesswire.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 37Low-dose Emicizumab prophylaxis in severe haemophilia A patients –a retrospective study bringing new hope for our patients. - 4 month(s) ago
– Low-dose Emicizumab can potentially offer a cost-effective treatment option in PwHA especially in developing countries. We evaluated its efficacy and safety when given for six months compared to low-dose Factor VIII prophylaxis in this chart review.
Source: www.jthjournal.orgCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 1Gene Therapy | Gene Therapy for Hemophilia | Gene Therapy Research - 4 month(s) ago
ISTH & the global hemophilia gene therapy experts have developed educational resources to address needs providing you a front seat to learn about hemophilia.
Source: GeneTherapy.ISTH.orgCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 38The story of Stephen Christmas and Christmas disease - 4 month(s) ago
How a boy called Stephen Christmas gave his name, voice and life to the fight against blood disease
Source: www.readersdigest.co.ukCategories: General Medicine News, Hem/OncsTweet
There is a great review article on gene therapy for #hemophilia B in the current issue of @BloodAdvances. I particularly liked the section on Features of Special Interest. The PDF is free. https://t.co/xSXQ9XBnSk