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Musculoskeletal abnormalities are congenital malformations or deformities of the musculoskeletal system that occur in isolation or as part of a syndrome.
In the LUMINA-1 trial for fibrodysplasia ossificans progressiva, garetosmab, an activin A monoclonal antibody, did not lead to significant changes in heterotopic ossification lesion activity in pre-existing lesions in period 1. Garetosmab prevented the formation of new lesions in both periods 1 and 2.
Ganesan et al. examine electronic health record data from over 100,000 patients with Dupuytren’s disease, a condition characterized by contracture of the hand, to look at its association with diabetes. They report a higher prevalence of Dupuytren’s disease in type 2 than in type 1 diabetes and in patients with HbA1c levels within the diabetic range.
Despite the transformative potential of treatments for systemic autoinflammatory diseases, these conditions remain underfunded and understudied. Urgent, coordinated action among stakeholders is needed to overcome regulatory and research barriers, as is innovation and advocacy for the development of effective therapies for these rare diseases.
The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci of expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models of myotonic dystrophy type 1.
Immune checkpoint inhibitors, which are used to treat many types of cancer, can cause syndromes similar to rheumatic diseases known as immune-related adverse events (irAEs). In 2020, several studies illustrated the clinical heterogeneity of rheumatic irAEs and highlighted their substantial effect on morbidity and mortality.
Jimmy Tang, from Newcastle Upon Tyne, is a dental associate in Peterlee and also a corrective exercise specialist, level 3 personal trainer and L4 sports massage therapist. An ex-back pain sufferer, Jimmy has a special interest in lower back pain and postural dysfunction.
The US Food and Drug Administration approved a muscular-dystrophy drug against the scientific advice of its own staff and advisors. Despite leadership's attempts to downplay the controversy, doubts now surround standards for accelerated approval.
