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Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints

- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose Dependent 27% Mean Splicing Correction Across All Patients in the 5.4 mg/kg Cohort at 3 Months - - DYNE-101 Showed Improvement in Myotonia,.

United statesStacy nartkerWildon farwellAmy reillyJohn coxExchange commissionDrug administrationMyotonic dystrophy health indexEuropean medicines agencyDyne therapeutics incDose dependentMean splicing correction across all patientsMuscle strengthTimed function testsReported outcomesMean unadjusted

Dyne Therapeutics Announces New Clinical Data from ACHIEVE

- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose Dependent 27% Mean Splicing Correction Across All Patients in the 5.4 mg/kg Cohort at 3 Months - .

United statesAmy reillyJohn coxStacy nartkerWildon farwellExchange commissionDyne therapeutics incEuropean medicines agencyDrug administrationMyotonic dystrophy health indexDose dependentMean splicing correction across all patientsMuscle strengthTimed function testsReported outcomesMean unadjusted

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights
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Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1

Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1
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San diegoUnited statesSarah boyceGeoffrey grandeNavjot raiAvidity biosciences incExchange commissionEuropean medicines agencyDrug administrationAvidity biosciencesAntibody oligonucleotide conjugatesBreakthrough therapyOrphan drugFast trackOrphan designationMyotonic dystrophy type

Myotonic Dystrophy Pipeline Assessment 2024 | In-Depth Insights Into The Emerging Drugs, Latest FDA Approvals, Clinical Trials, Ter, Key Companies

Myotonic Dystrophy Pipeline Assessment 2024 | In-Depth Insights Into The Emerging Drugs, Latest FDA Approvals, Clinical Trials, Ter, Key Companies
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Nexien biopharmaCompanies in myotonic dystrophy therapeutics marketLocana incRoute of administrationDrug administrationAdministration roPepgen corporationKey companiesSyros pharmaceuticalsDystrophy pipelineMyotonic dystrophy marketHarmony biosciencesAvidity biosciencesDyne therapeuticsEntrada therapeuticsArthex biotech