USF Health researchers find experimental drug may protect the heart in Duchenne muscular dystrophy - University of South Florida

A ten-person trial combined gene therapy with camera-equipped goggles to test an experimental treatment for advanced retinitis pigmentosa. Seven participants gained light sensitivity, while four of eight completing behavioral tests showed consistent improvements across tasks. The small safety-focused study found modest visual gains, with substantial uncertainty about efficacy and no restoration of normal sight. A notebook or small box on a table became easier to detect for some people whose retinal disease had severely damaged their sight. An experimental treatment gave surviving retinal nerve cells a new ability to respond to light. Special goggles supplied the signals those cells needed to convey limited visual information. The ten-person PIONEER trial, reported in The New England Journal of Medicine, primarily assessed safety. Seven participants showed increased light sensitivity after treatment, and four showed consistent gains across several behavioral tests. Improvements varied considerably, and the approach did not restore normal or high-resolution vision. Diagram of the new therapy. ① Gene therapy makes retinal cells light-sensitive.

Sixty Surrey fire fighters tapped into a generous public and collected $25,000 for Muscular Dystrophy Canada (MDC) this year, bringing the Local’s lifetime donations to the charity to $266,000. The effort earned the Surrey Fire Fighters Charitable Society the 2026 Dr. David Green Award for Fire Department of the Year, which recognizes its fundraising and longstanding support for the charity.