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New Advancement Toward The First Primary Endpoint Qualification Of A Digital Endpoint For Duchenne

Current methods for assessing ambulatory function in patients with Duchenne muscular dystrophy do not fully reflect how they experience DMD in their.

Region flamandeBruxelles capitaleFrance generalRobert debrRoxana donisa dreghiciLaurent servaisCarl morrisDonisa dreghiciHead of clinical developmentLouvain medical schoolNovo nordiskFranklin pierce collegeUniversity of medicineDepartment of physiologyEuropean medicines agencyInstitute of myology

Avidity Biosciences Reports Third Quarter 2022 Financial Results and Recent Highlights

Company has three distinct rare disease programs in clinical development - myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy.

United statesSan diegoNavjot raiKathleen gallagherMike macleanEli lillySarah boyceCompany lillyInternational hybrid annual congressDrug administrationPrnewswire avidity biosciences incExchange commissionDevelopment rd expensesWorld muscle societyAvidity biosciences incAntibody oligonucleotide conjugates

Avidity Biosciences Reports Third Quarter 2022 Financial Results and Recent Highlights

/PRNewswire/ Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody.

United statesSan diegoNavjot raiKathleen gallagherMike macleanEli lillySarah boyceCompany lillyInternational hybrid annual congressDrug administrationPrnewswire avidity biosciences incExchange commissionDevelopment rd expensesWorld muscle societyAvidity biosciences incAntibody oligonucleotide conjugates

BridgeBio Pharma, Inc Reports Third Quarter 2022 Financial Results and Business Update

03.11.2022 - - Reported positive preclinical data for its next-generation KRASG12C GTP/GDP dual inhibitor development candidate, BBO-8520, and for its novel PI3Kα:RAS breaker mechanism in late lead optimization - Reported positive updated 12-month Phase 2 data .

United statesMedison pharmaVikram baliBrian stephensonNeil kumarBristol myers squibbFrank mccormickAmgen lumakrasEndocrine societyIsrael ministry of healthBridgebio pharma incEuropean unionWorld muscle societyCompany phaseInternational hybrid annual congressOrigin biosciences inc

NS Pharma Reports on Results of VILTEPSO® (viltolarsen) injection After Four Years of Treatment in Open-Label Extension Trial in Duchenne Muscular Dystrophy

/PRNewswire/ NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru.

United statesTsugio tanakaToru nakaiLeslie magnusNs pharma incPrnewswire ns pharma incNippon shinyaku co ltdInternational hybrid annual congressWorld muscle societyNippon shinyakuNova scotiaVice presidentImportant safetyMedical informationRns pharmaMedical pharmaceuticals

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