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- FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type IaThe U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen…
- Intellia finds genetic marker linked to liver enzyme spikes in CRISPR ATTR amyloidosis trialIntellia Therapeutics announced that analysis of its ongoing CRISPR gene-editing trial for transthyretin (ATTR) amyloidosis has identified a common genetic variant among participants who experience…Endpoints · 14d agoNegative readoutNegative