ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK
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ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK

  An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…

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This Treatment for Multiple Sclerosis Could be on its Way to Approval in the EU
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This Treatment for Multiple Sclerosis Could be on its Way to Approval in the EU

According to a story from finanznachricten.de, the drug company Novartis recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive…

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Life as a Parent of a Child With Glycogen Storage Disease: A Rare Disease Story From the Czech Republic
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Life as a Parent of a Child With Glycogen Storage Disease: A Rare Disease Story From the Czech Republic

When your child is constantly at risk of hypoglycemia and their health and life is threatened in the case of any major problem, you have no other choice but to…

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