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CABA-201 gets FDA orphan drug status as scleroderma treatment

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Cabaletta Bio’s cell therapy CABA-201 as a treatment for adults with hard-to-treat systemic sclerosis (SSc), or scleroderma. Orphan drug designation is intended to support the accelerated development of investigational treatments for rare diseases, defined as…

Frequency of muscle disease in SSc shows need for biomarkers: Study

Muscle disease is common in people with scleroderma and is associated with inflammation and specific end-organ involvement, according to an Australian study. The findings underscore the “clinical, functional, and prognostic importance of simple biomarkers ” for identifying SSc-related muscle disease, the study’s researchers wrote in “Proximal weakness…