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Related Concept Videos

Pulmonary Function Tests01:25

Pulmonary Function Tests

779
Pulmonary Function Tests (PFTs)
Pulmonary Function Tests are crucial diagnostic tools for assessing respiratory function, particularly in patients with chronic respiratory disorders. They comprehensively evaluate lung volumes, ventilatory function, breathing mechanics, diffusion, and gas exchange. These tests help diagnose pulmonary diseases and play a significant role in monitoring disease progression, evaluating disability, and assessing response to therapy.
PFTs involve using a spirometer, a...
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Correlations02:20

Correlations

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Correlation means that there is a relationship between two or more variables (such as ice cream consumption and crime), but this relationship does not necessarily imply cause and effect. When two variables are correlated, it simply means that as one variable changes, so does the other. We can measure correlation by calculating a statistic known as a correlation coefficient. A correlation coefficient is a number from -1 to +1 that indicates the strength and direction of the relationship between...
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Correlation and Causation01:27

Correlation and Causation

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Statistical tests can calculate whether there is a relationship, or correlation, between independent and dependent variables. An indirect relationship of the variables signifies a correlation, while a direct relationship shows causation. If it is determined that no connection exists between the variables, then the correlation is a coincidence.
Correlation versus Causation
If the dependent variable increases or decreases when the independent variable increases, there is a positive or negative...
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Correlation01:09

Correlation

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In statistics, two variables are said to be correlated if the values of one variable are associated with the other variable. Depending on the relationship between two variables, correlation can be of three types– positive correlation, negative correlation, and zero correlation.
Two variables, for example, a and b, are said to be positively correlated if both variables move in the same direction. In other words, a positive correlation exists between two variables, a and b, if:
14.8K
Correlation and Regression00:53

Correlation and Regression

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In statistics, correlation describes the degree of association between two variables. In the subfield of linear regression, correlation is mathematically expressed by the correlation coefficient, which describes the strength and direction of the relationship between two variables. The coefficient is symbolically represented by 'r' and ranges from -1 to +1. A positive value indicates a positive correlation where the two variables move in the same direction. A negative value suggests a...
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Coefficient of Correlation01:12

Coefficient of Correlation

8.5K
The correlation coefficient, r, developed by Karl Pearson in the early 1900s, is numerical and provides a measure of strength and direction of the linear association between the independent variable x and the dependent variable y.
If you suspect a linear relationship between x and y, then r can measure how strong the linear relationship is.
What the VALUE of r tells us:
The value of r is always between –1 and +1: –1 ≤ r ≤ 1.
The size of the correlation r indicates the...
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Updated: Jan 24, 2026

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Pulmonary function and clinical correlation in DMD.

Oscar H Mayer1

  • 1Perelman School of Medicine at The University of Pennsylvania, Division of Pulmonary Medicine, The Children's Hospital of Philadelphia, 3501 Civic Center Boulevard, Philadelphia, PA 19104, United States.

Paediatric Respiratory Reviews
|May 28, 2019
PubMed
Summary
This summary is machine-generated.

Duchenne muscular dystrophy (DMD) is an inherited myopathy caused by dystrophin gene defects. Advances in pharmacotherapy now target dystrophin production and downstream effects, offering new treatment avenues.

Keywords:
Duchenne muscular dystrophyDystrophinPharmacological therapiesRespiratory failure

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Area of Science:

  • Genetics and Molecular Biology
  • Neurology
  • Pharmacology

Background:

  • Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy.
  • It results from mutations in the dystrophin gene on the X chromosome, leading to dysfunctional dystrophin protein.
  • Recent advancements have expanded therapeutic strategies beyond corticosteroids.

Purpose of the Study:

  • To explore the respiratory progression in Duchenne muscular dystrophy.
  • To correlate functional and clinical aspects of respiratory decline.
  • To review current and developmental pharmacotherapies for DMD.

Main Methods:

  • Literature review of DMD progression and pharmacotherapy.
  • Analysis of functional and clinical correlations in respiratory decline.
  • Synthesis of information on symptomatic treatments and support.

Main Results:

  • DMD involves progressive respiratory muscle weakness.
  • Pharmacotherapies aim to restore dystrophin production or mitigate downstream effects.
  • Symptomatic treatments and support are crucial for managing respiratory complications.

Conclusions:

  • Understanding respiratory progression in DMD is key for timely intervention.
  • Novel pharmacotherapies offer potential for disease modification.
  • Comprehensive management strategies are essential for improving outcomes in DMD patients.