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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

Neoleukin Therapeutics : Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome - Form 8-K -December 19, 2023 at 07:34 am EST

Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome Two pediatric patients with Rett.

Baylor college of medicineUnited statesNew yorkMelissa forstBernhard suterRachel mcminnDavid rosenDrug administrationNeoleukin therapeutics incSecurities exchangeExchange commissionNeurogene incNeurogene doses first patientsFemale pediatric patientsExpression attenuationConstruct tuning

2 US Girls Become World s First to Get Gene Therapy for Genetic Brain Disorder – Eastern Mirror

2 US Girls Become World s First to Get Gene Therapy for Genetic Brain Disorder – Eastern Mirror
easternmirrornagaland.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from easternmirrornagaland.com Daily Mail and Mail on Sunday newspapers.

United statesBaylor college of medicineRachel mcminnBernhard suterHuda zoghbiDan duncan neurological research instituteBaylor collegeHoward hughes medical instituteTexas childrenAssociate professor

2 Girls Become World s First To Get Gene Therapy For Brain Disorder

2 Girls Become World s First To Get Gene Therapy For Brain Disorder
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United statesBaylor college of medicineHuda zoghbiRachel mcminnBernhard suterBaylor collegeHoward hughes medical instituteDan duncan neurological research instituteBecome worldTexas childrenAssociate professor

2 US girls become world s first to get gene therapy for genetic brain disorder

A team of US doctors have in a world first delivered a novel gene therapy to treat Rett syndrome a rare genetic brain disorder in two girls. They became the first children worldwide to receive the promising treatment as part of the first-in-human Phase I/II trial of a new investigational gene therapy, NGN-401, developed by US-based Neurogene.

Baylor college of medicineUnited statesBernhard suterHuda zoghbiRachel mcminnBaylor collegeHoward hughes medical instituteDan duncan neurological research instituteTexas childrenAssociate professorRett syndromeGene therapyBrain disorderHuman trailBrain problem