Corticosteroids may improve or stabilize motor function in Fukuyama congenital muscular dystrophy

This is shown by a Japanese prospective study carried out openly in 9 children with an average age of 8.1±2.14 years. One had a moderate form of Fukuyama congenital muscular dystrophy (able to walk), another a severe form (inability to hold his head up) and the other seven could sit up unaided (classic form).

Alternate day treatment with prednisolone (0.5 mg/kg for 2 patients, 0.7 mg/kg for one patient, and 1 mg/kg for the remaining six) was initiated when motor function began to decline.

After 8.86 ± 3.05 months of treatment,

  • the overall score of the Gross Motor Function Measure (GMFM) scale improved by 1.23 ± 1.1 points in eight of the nine children, even if the scores on the various dimensions of the GMFM (lying down and rolling over, sitting, crawling and kneeling, standing, walking, running and jumping) were heterogeneous (with improvement in one or more dimensions and deterioration and/or stabilization of others),
  • note that an improvement in the score of the “Lying and rolling” dimension accompanied the decrease in the overall score of the ninth child,
  • the side effects of corticosteroid therapy (irritability in 4 cases, one of whom also presented with an increase in appetite and a Cushingoid facies) never justified its discontinuation.


Efficacy of steroid therapy for Fukuyama congenital muscular dystrophy. Murakami T, Sato T, Adachi M, et al. Sci Rep. 2021 Dec 20;11(1):24229.