The most commonly diagnosed form of muscular dystrophy, Duchenne is a genetic disease that affects mostly boys and causes progressive muscle weakness over time. Its early physical signs tend to be ...

Macular dystrophies (MDs) consist of a heterogeneous group of disorders that are characterised by bilateral symmetrical central visual loss. Advances in genetic testing over the last decade have led ...

Almost a year after buying Kate Therapeutics, Novartis has scooped up another San Diego-based muscle dystrophy biotech in one of the biggest acquisitions of 2025 so far. The Swiss pharma is paying $12 ...

The Financial Industry Regulatory Authority (FINRA)’s Board of Governors has approved a major overhaul of its pattern day trading (PDT) rules, marking a critical shift in how active retail trading ...

Background/aims Inherited retinal dystrophies (IRD) are a group of predominantly monogenic disorders which have genetically heterogeneous origins and display wide clinical phenotypic heterogeneity.

Christine McSherry is a registered nurse and founder of Jett Foundation. On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

Geographic atrophy is diagnosed with a dilated eye exam and imaging testing such as retinal fundus photography (a color image of the retina) or autofluorescence imaging (which shows where geographic ...