People and families living with rare diseases and disability have access to a suite of updated resources to help navigate...

The Save Our Sons Duchenne Foundation is deeply saddened by the passing of our dear friend and long-standing ambassador, Gary...

Save Our Sons Duchenne Foundation has joined the rare disease community at NSW Parliament House for an event focused on...

Access to care and support can change lives for families living with Duchenne muscular dystrophy. This year’s World Duchenne Awareness...

More than 500 people gathered in Sydney to support people living with Duchenne and Becker muscular dystrophy at Save Our...

 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...