Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy

According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement tool for Duchenne muscular dystrophy symptoms, to provide a quantitative analysis of patient data. In the future, the NSAA could be used in clinical trials to measure endpoints and patient outcomes.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.

About The NSAA

The NSAA is tailored to measure a patient’s ability to conduct a diverse array of physical tasks, such as rising from the floor, running, walking, hopping, climbing stairs, and jumping. This approach allows the assessment to deliver a greater degree of detail in characterizing a patient’s degree of disease progression in comparison to prior tools.

The researchers looked at data from 395 Duchenne muscular dystrophy disease patients. The scientists were able to categorize the patients into different groups by rates of disease progression, which highlights the variation of presentation that can appear in the disorder. The researchers were also able to provide a risk assessment in order to predict a patient’s future rate of progression or other functional changes. 

The results from this study demonstrate the capability of the NSAA when used both as an endpoint measure and as a tool for predicting disease progression.

Check out the original study, published in the journal PLOS One, here.

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