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Updated: Jan 30, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
09:18

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Published on: January 12, 2019

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Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

Anne M Connolly1,2, Craig M Zaidman1,2, Paul T Golumbek1,2

  • 1Department of Neurology, Washington University School of Medicine in Saint Louis, St Louis, Missouri, 63110, USA.

Muscle & Nerve
|February 2, 2019
PubMed

Insights

Twice-weekly glucocorticosteroids (GC) in young boys with Duchenne muscular dystrophy (DMD) improved gross motor function. While generally well-tolerated, excessive weight gain was noted, requiring further investigation for long-term feasibility.

Area of Science:

  • Pediatric Neurology
  • Genetics and Genetic Diseases
  • Clinical Trials

Background:

  • Glucocorticosteroids (GC) are established treatments for slowing muscle weakness in Duchenne muscular dystrophy (DMD).
  • Early intervention in DMD is crucial for optimizing long-term outcomes.
  • The safety and efficacy of GC in very young DMD patients require further evaluation.

Purpose of the Study:

  • To assess the efficacy and tolerability of twice-weekly prednisolone in infants and young boys with DMD.
  • To compare motor development outcomes in treated infants/boys with a historical control cohort.

Main Methods:

  • A 1-year, open-label, multisite trial involving 25 infants/young boys (0.4-2.4 years) with DMD receiving twice-weekly prednisolone.
  • Motor development was assessed using the Bayley III Scales of Infant Development (Bayley-III), specifically the Gross Motor Scaled Score (GMSS).
  • Outcomes were compared to a historical control cohort (HCC) of untreated boys.

Main Results:

  • Treated boys showed an average gain of 0.5 points in GMSS, while the HCC declined by 1.3 points (P=0.03).
  • All participants maintained linear growth, and no Cushingoid features were observed.
  • Excessive weight gain occurred in 56% of treated boys.

Conclusions:

  • Twice-weekly GC administration appears well-tolerated in infants and young boys with DMD and demonstrates a positive impact on gross motor function.
  • Excessive weight gain is a notable side effect that warrants monitoring.
  • Further long-term studies are needed to confirm the feasibility of early GC initiation in this population.
Abstract

Keywords:
Bayley-III Scales of Infant and Toddler DevelopmentDMDDuchenne muscular dystrophyGCinfantstwice-weekly glucocorticosteroid

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