News

  • Wave Life Sciences Shares 48-Week Data from FORWARD-53 Trial of WVE-N531 in Duchenne Muscular Dystrophy

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    Dear Members of the Duchenne community,We are excited to share positive data following 48 weeks of dosing in the FORWARD-53 clinical trial eval...

  • Duchenne Care Conference 2025

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    The World Duchenne Organization is thrilled to announce that registrations are now open for the Duchenne Care Conference 2025. The online confe...

  • Sarepta Therapeutics Shares Safety Update on ELEVIDYS

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    CAMBRIDGE, Mass., March 18, 2025 -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, share...

  • Dyne Therapeutics Announces New Long-Term Clinical Data

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    Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating U...

  • Edgewise Therapeutics Topline results from phase 2 Becker Canyon trial of Sevasemten

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     - Trial met primary endpoint of reduction in circulating levels of creatine kinase (CK), a biomarker associated with skeletal muscle damage, i...

  • Update to the Duchenne Community from Percheron Therapeutics on Progress with Avicursen

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    Dear Members of the Duchenne Community, Today, Percheron Therapeutics, a public company registered in Australia and focused on developing treat...

  • No Life Insurance Discrimination For Those With a Genetic Condition

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    Australian Government bans genetic discrimination in life insurance: A big win for preventive health Following the recommendations of a r...

  • Final Letter for Philips Recall

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    Philips after consultation with TGA is conducting an Urgent Product Defect Correction for the Philips BiPAP A40 Pro Ventilator, BiPAP A40 E...

  • Update to Urgent Product Defect Alert TGA Ref RC-2024-RN-00257-1- Philips Ref 2023-CC-SRC-039

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    Philips after consultation with TGA is conducting an update to the Urgent Product Defect Alert of Philips BiPAP A30, BiPAP A30 EFL, BiPAP A...

  • Pfizer Phase 2 Daylight Study Update

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  • Pfizer gene therapy clinical trials commence in Australia

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    Pfizer gene therapy clinical trials commence in Australia! The first Australian boy has been screened as part of the Pfizer gene therapy clinica...

  • “New Frontiers” Launched

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    The long awaited Federal Parliamentary Inquiry report into Approval Process for New Drugs and Novel Technologies has now been released. The reco...