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BridgeBio Pharma Presents Updated Encouraging Clinical and Biomarker Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

- Following treatment, the N-acetylaspartate levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s. | May 22, 2023

United statesGenevieve laforetFlorian eichlerBio pharma incBridgebio pharma incDrug administrationEuropean unionExchange commissionEuropean medicines agencySafety monitoring committeeSecurities exchangeGross motor functionBio pharmaAspa therapeuticsMassachusetts general hospitalRare neurological disease

A young boy s nightmare diagnosis, and the $3 million one-time treatment that will likely save his life

The groundbreaking treatment is one of six gene therapies cleared since 2017 that have transformed the outlook for disorders once considered hopeless and also renewed questions about how the health care system can afford such breathtakingly expensive medicines.

United statesTufts medical centerNew yorkUniversity of pennsylvaniaMichael shermanJess rowlandsFlorian eichlerAdam hessDarcy grayConner hessEdward kayeChristine duncanDaniel ollendorfGlynda jacksonAndrew obenshainEzekial emanuel

NTSAD to honor Guangping Gao for lifetime work on Canavan disease, as gene therapy trial shows promise

Guangping Gao, PhD, will be honored by the National Tay-Sachs & Allied Diseases Association for his work in identifying the Canavan gene, revitalizing gene therapy, and his many accomplishments leading to potential treatments for Canavan disease.

United statesBio pharmaFlorian eichlerGuangping gaoDrug administrationHarvard medical schoolHospital centerPenelope booth rockwell professor in biomedical researchNational tay sachs allied diseases associationEuropean unionLi weibo institute for rare diseases researchHorae gene therapy centerNational tay sachsAllied diseases associationPenelope booth rockwell professorBiomedical research

BridgeBio Pharma Inc (BBIO) Presents Updated Positive Data from its BBP-812 Canavan Disease Gene Therapy Program

BridgeBio Pharma Inc (BBIO) Presents Updated Positive Data from its BBP-812 Canavan Disease Gene Therapy Program
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Bridgebio pharmaFlorian eichlerGenevieve laforetGuangping gaoCanavan foundationNational tay sachsClinical development at aspa therapeuticsCanavan research illinoisChild neurology societyBridgebio pharma incDiseases associationBio pharmaAspa therapeuticsBridgebio gene therapyNeurology societyPrincipal investigator

BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease

BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease
streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.

United statesFlorian eichlerGenevieve laforetOrren alpersteinCanavan foundationDrug administrationResearch dayLeukodystrophy serviceExchange commissionBridgebio pharma incEuropean unionBio pharma incSecurities exchangeEuropean medicines agencyPharma incWall street