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Wave Life Sciences : Potent, Durable mRNA Knockdown in Extrahepatic Tissues Using siRNAs With Novel Phosphoryl Guanidine Backbone Variants

Wave Life Sciences : Potent, Durable mRNA Knockdown in Extrahepatic Tissues Using siRNAs With Novel Phosphoryl Guanidine Backbone Variants
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Rett syndrome

Neurogene : ASGCT NGN-401 Clinical Safety Poster

Preliminary Safety Results from the Ph1/2 Study of NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome Suter B1, Lieberman D2, Benke T3,.

United statesNew yorkBaylor college of medicineMonroe carell jrNeurogene incDepartment of pediatricsHarvard medical schoolAmerican society of geneDepartment of pediatrics neurologyVanderbilt university medical centerUniversity of colorado school medicineDepartment of neurologyPreliminary safety resultsNovel regulated gene therapyRett syndromeTexas children

Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome during ASGCT Annual Meeting

NGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-up No signs or symptoms of overexpression toxicity, including in one patient with a mild.

United statesBernhard suterRachel mcminnAmerican society of geneCompany annual report on formExchange commissionNeurogene incAmerican societyCell therapyChief executive officerRett syndromeClinical global impression severityMedical directorBlue bird circle rett centerTexas childrenAssociate professor

LottieFiles Raises Strategic Investment from Webflow Ventures

LottieFiles Raises Strategic Investment from Webflow Ventures
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Administration roRett syndrome

Uveal Melanoma Market Is Expected to Showcase a Significant

Uveal Melanoma Market Is Expected to Showcase a Significant
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Administration roRett syndrome