Muscle diseases cover a diverse group of disorders that, in most cases, are hereditary. The
rarity of the individual muscle diseases provides a challenge for researchers when wanting …

Clinical trials with treatments inhibiting myostatin pathways to increase muscle mass are
currently ongoing in spinal muscular atrophy. Given evidence of potential myostatin pathway …

Tubular aggregate myopathy (TAM) and Stormorken syndrome (STRMK) are clinically
overlapping disorders characterized by childhood-onset muscle weakness and a variable …

The identification of biomarkers for spinal muscular atrophy is crucial for predicting disease
progression, severity, and response to new disease-modifying therapies. This study aimed …

Introdução: A Atrofia Muscular Espinhal (AME) é uma doença neurodegenerativa
autossômica recessiva, caracterizada pela degeneração progressiva e irreversível dos …