PTC Therapeutics Provides Regulatory Update on Translarna™  February 12, 2026  WARREN, N.J., Feb. 12, 2026 /PRNewswire/ -- PTC Therapeutics, Inc....

Friday, 28 November 2025, 9AM - 5:00PM Level 3, 48 Flemington Road, Parkville, Vic A one-day professional learning workshop for...

First returning adult patient from Phase 1b study recently dosed; additional returning patients being scheduled 11-month open-label study will evaluate...

Genetics Webinar Series - Events WEBINAR 1 Could It Be Genetic? If you've ever wondered whether there might be a...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 21, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued...

– New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings –...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 15, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided...

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

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

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

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

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

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

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

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

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

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

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

Save Our Sons Duchenne Foundation has just written a submission on behalf of our community to the Senate Standing Committee...