Muscular sarcoidosis is a rare disease characterised by non-caseating granulomas in muscle tissue. We report a woman in her late 70s with progressive lower-limb weakness, in whom ultrasonography ...

WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

Many patients are given a different diagnosis initially, including lupus erythematous and undifferentiated connective tissue disease. It is of paramount importance to determine the organ (i.e. skin, ...

On January 6, 2025, the U.S. Food and Drug Administration (“FDA”) and the Office for Human Research Protections (“OHRP”) issued a joint draft guidance on including tissue biopsies in clinical trials ...