Corticosteroid treatment in early-onset lamin A/C related muscular dystrophies


Corticosteroid therapy has shown a benefit in Duchenne muscular dystrophy especially on delaying loss of ambulation, reducing the incidence and severity of scoliosis and the age at respiratory insufficiency. It is also beneficial in myositis. As the muscle biopsies of patients with lamin A/C mutations can have prominent inflammatory component, corticosteroids have been empirically used in several patients in the past. We present a series of 27 patients with early-onset muscular dystrophy associated with lamin A/C mutations, 21 of them are part of an international multicenter retrospective natural history study.


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