Related Experiment Video

Updated: Oct 16, 2025

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

10.5K

Health Profile of Preterm Males With Duchenne Muscular Dystrophy

Aida Soim1, Bailey Wallace2,3, Nedra Whitehead4

  • 11094New York State Department of Health, Albany, NY, USA.

Journal of Child Neurology
|October 22, 2021
PubMed

Insights

Preterm birth may impact Duchenne muscular dystrophy (DMD) progression, particularly respiratory interventions. While overall disease course is similar, preterm males with DMD may experience earlier pulmonary function decline.

Area of Science:

  • Neurology
  • Pediatrics
  • Genetics

Background:

  • Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disorder.
  • Preterm birth is a significant factor influencing neonatal health outcomes.
  • Limited data exists on the specific health profiles of preterm males with DMD.

Purpose of the Study:

  • To characterize the health profile and disease progression in preterm males with Duchenne muscular dystrophy.
  • To compare clinical milestones and treatment patterns between preterm and full-term males with DMD.
  • To identify potential differences in disease impact related to prematurity.

Main Methods:

  • Retrospective cohort study utilizing a population-based surveillance system.
  • Analysis of major clinical milestones: ambulation cessation, assisted ventilation, and left ventricular dysfunction.
  • Kaplan-Meier survival curves and Cox proportional hazards modeling were employed.

Main Results:

  • Preterm males with DMD had an 87% higher adjusted risk for respiratory interventions compared to full-term males.
  • Adjusted risks for ambulation cessation and left ventricular dysfunction were modestly elevated but not statistically significant.
  • No significant difference was observed in the initiation of corticosteroid therapy between preterm and full-term males with DMD.

Conclusions:

  • The overall disease course of Duchenne muscular dystrophy appears similar between preterm and full-term males.
  • Pulmonary function may be affected earlier in preterm males with DMD, necessitating closer monitoring.
  • Prematurity is associated with an increased risk of respiratory complications in Duchenne muscular dystrophy.

Keywords:
Duchenne muscular dystrophychildren epidemiologypediatricpreterm

More Related Videos

Assessing Functional Performance in the Mdx Mouse Model10:32

Assessing Functional Performance in the Mdx Mouse Model

Published on: March 27, 2014

32.9K
Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice08:13

Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Published on: April 10, 2019

6.2K

Related Concept Videos

Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
2.1K
Disorders of the Male Reproductive System01:20

Disorders of the Male Reproductive System

Men's health issues are increasingly recognized as significant, with several conditions posing common threats. Among these, testicular cancer is especially prevalent in younger men, particularly those aged 20 to 35 years. The disease often manifests as a painless mass in the testicles, sometimes accompanied by a sensation of heaviness or a dull ache.
Prostate disorders are another major concern. These conditions can impair urinary flow due to the prostate's location around the urethra....
2.4K
Cardiomyopathy III: Hypertrophic Cardiomyopathy01:29

Cardiomyopathy III: Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy, or HCM, is an autosomal dominant genetic disorder characterized by asymmetric left ventricular hypertrophy without ventricular dilation. It is more common in men and is typically diagnosed in young, athletic adults.EtiologyHCM is primarily genetic and is caused by mutations in genes encoding sarcomeric proteins. Researchers have identified over 1400 mutations across at least 11 different genes. Among these, the most frequently occurring mutations are found in the...
94
Sex-linked Disorders01:43

Sex-linked Disorders

Like autosomes, sex chromosomes contain a variety of genes necessary for normal body function. When a mutation in one of these genes results in biological deficits, the disorder is considered sex-linked.
103.7K
Pedigree Analysis01:35

Pedigree Analysis

Overview
86.1K
Disorders of the Skeletal Muscle01:28

Disorders of the Skeletal Muscle

The clinical conditions affecting the skeletal muscle tissue are broadly categorized as musculoskeletal and neuromuscular disorders.
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
1.3K