Weekly Spotlight - 14.11.24

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In the News

Archer Sverdrup: Transforming Lives Through Power Soccer and Community Support

Archer Sverdrup, a St. Louis native, co-founded Everything Early Onset, a support group for early-onset FSHD sufferers. Living with FSHD since birth, Archer fosters community and understanding. They plan to pursue a doctorate in occupational therapy, aiming to make a positive impact on the disabled community.

Del-brax Trial Offers Hope for FSHD Patients

A new Phase 1/2 trial of del-brax is enrolling patients with FSHD, aiming to gather biomarker data for potential FDA accelerated approval. Del-brax targets the DUX4 protein, believed to cause FSHD symptoms. The trial, spanning the US, UK, and Canada, seeks to offer hope for those without treatment options.

DUX4 Regulation in Muscular Dystrophy: p38's Role Explored

The study explores the role of p38 MAP kinase in regulating DUX4 expression in facioscapulohumeral muscular dystrophy (FSHD). It reveals that p38 is crucial for early DUX4 expression but not in later stages. This finding suggests potential therapeutic strategies, offering hope for improved treatments in the future.

Avidity Biosciences Advances FORTITUDE Trial for Muscular Dystrophy Treatment

Avidity Biosciences has launched a biomarker cohort in the FORTITUDE trial for AOC 1020, targeting facioscapulohumeral muscular dystrophy. The trial shows promising reductions in DUX4-regulated genes and improved muscle function. AOC 1020 is well-tolerated, offering hope for patients without current treatment options.