Very Young Boys with DMD May Have Measurable Early Signs of Motor Skill Delay, Study Suggests

Very young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for the early detection of DMD and for evaluating how treatments affect their development as opposed to the natural course of the disease, a study shows.

Findings of the study, “Early signs of gross motor delay in very young boys with Duchenne muscular dystrophy,” were presented in a poster session at the 2019 American Academy of Neurology (AAN) annual meeting, taking place through May 10 in Philadelphia.

As therapies and treatments for DMD continue to evolve, it’s becoming increasingly clear that early diagnosis and earlier treatment can lead to better outcomes. Typically, DMD is diagnosed in children at between 3 and 5 years of age.

“The differentiation between healthy infants and those with DMD has not been established in infants and young children,” the researchers wrote.

It’s also important to have measurements that are actually reflective of the state of disease — that is, clinicians and researchers need to be sure that the tests they use to measure, for example, motor skills, are actually assessing this in a way that allows both distinguishing between children with and without DMD and tracking how treatments improve (or don’t improve) these skills in children with DMD.

To this end, researchers recruited 114 boys with DMD (ages from about 10 months to nearly 8 years old) and gave them three different tests of motor skills: the motor function part of the Bayley Scales of Infant and Toddler Development (third edition), the North Star Ambulatory Assessment (NSAA), and the 100-meter timed test.

All of these tests involve testing motor skills — for example, the time it takes for a child to run around some cones or climb a set of stairs. Since there is published data for what a “normal” score is for each of these tests, the researchers were able to compare the scores in their DMD group with these normal ones.

Their goal was both to determine whether these tests were effective measurements in DMD and to find out what score or range of scores in each test might be indicative of DMD.

All three tests were found to be effective at differentiating between boys with DMD and normally developing boys, with lower scores for DMD patients. For example, in the 100-meter timed test, the scores of boys with DMD were, on average, about half of the normal value.

“Difficulty with key skills were indicative of early gross motor involvement. The delay in gross motor skills remained present over time,” the authors wrote.

They also compared the results of patients on steroids with those of patients not on steroids, showing that patients treated with steroids had significantly higher scores on all outcomes.

“Clinicians can use the expected trajectories [Bayley-III, NSAA and 100 meter] to better counsel families on disease course,” the poster read. “The Bayley-III, NSAA and 100 m can be successfully implemented in the young DMD cohort and may be useful tools for clinical use and as trial outcome measures.”

Source : MD NEWS

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Public Health to us at MDCRC is about the community at large. The health of the community at different levels and when seen through different perspectives becomes a subject which is complete, holistic and is a melting pot for a plethora of technical disciplines. At MDCRC we do align with a goal of working for community health by working on modular projects related to Women and Child health, disease prevention, Health informatics, Community building and its associated research, Water resources, Arts, Indic Sciences, Cultural issues which are relevant to the contemporary generation. The style which we adopt is to focus on totally neglected aspects and form a working model towards a better situation. The need for this approach is to bring back the glory of humanity in general and which percolates to a healthier India.

The disorders in current focus are Orphan disorders, meaning disorders whose numbers are said to be less (in the Western diaspora) which neither doesn’t majorly impact the community’s currently used health metric nor does it provide a base for extensive drug research and its production. This view is only true until the numbers are low and also the way we look at Public health in general. There had been no concerted efforts in the domain of Orphan disorders in India. Mission of ours then, was to bring the science, the services pertaining to a particular problem statement and which thereby gives rise to an evolution of a model. This forges a way of approaching Health from a community standpoint thereby offering an impactful experience to all stake-holders. This is where the core culture MDCRC comes in where we believe that in Public Health domain in particular, the science and the associated services need to reach mankind at large without a number bias or number crunching.

With a background in Biochemistry and Genetics, Dr. Lakshmi, in 2006 had started working on genetic disorders with specific emphasis on community building.

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