Abstract Details

Title Early signs of gross motor delay in very young boys with Duchenne muscular dystrophy

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-068

Objective This study aims to 1) examine the utility of the Gross Motor portion of the Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), North Star Ambulatory Assessment (NSAA), and 100 Meter Timed Test (100m) for use in the very young Duchenne muscular dystrophy (DMD) cohort, 2) confirm differences in motor performance between young boys with DMD and typically developing peers and 3) document natural history and average values in young boys with DMD.

Background In the era of evolving therapies for the treatment of DMD, outcome measure selection is of upmost importance. Preclinical and early clinical trial data strongly suggest that early treatment will maximize benefits. With the historic average range of diagnosis between the ages of 3-5 years, the differentiation between healthy infants and those with DMD has not been established in infants and young children. Similarly, the magnitude of a change in development different from natural history to document improvements is not as well defined as the older cohorts.

Design/Methods 114 boys with a confirmed diagnosis of DMD, 0.8–7.9 years (Mean 4.7±1.5), were evaluated using the Bayley-III (N=46, 0.8–5.9 years), NSAA (N=107, 2.5-7.9 years) and 100m (N=74, 3.5–7.9 years) during clinic visits.

Results Bayley-III scaled scores were lower in boys with DMD compared to published controls (Mean 4.7 ± 1.9; typical peers 10 ± 3). 100m times were significantly lower than age/size matched peers across the age range (Mean 53.4% of predicted). Difficulty with key skills were indicative of early gross motor involvement. The delay in gross motor skills remained present over time.

Conclusions

Gross motor delay can be measured in infants and young boys with DMD using the Bayley-III, NSAA, and 100m. Reference values by age and steroid regimen will be presented to document the natural history.

Authors/Disclosures
Natalie F. Miller, PT PRESENTER	Dr. Miller has received personal compensation for serving as an employee of ATOM International. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casimir.
Lindsay N. Alfano, PT (Nationwide Children'S Hospital)	Ms. Alfano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ATOM International, Ltd (Amicus Therapeutics, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics). Ms. Alfano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Novartis Gene Therapies. The institution of Ms. Alfano has received research support from Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Audentes Therapeutics/Astellas Gene Therapies. Ms. Alfano has received intellectual property interests from a discovery or technology relating to health care.
	No disclosure on file
	No disclosure on file
	No disclosure on file
Chang-Yong Tsao, MD, FAAN (Nationwide children's hospital)	No disclosure on file
Megan A. Waldrop, MD (Nationwide Children's Hospital)	Dr. Waldrop has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sarepta Therapeutics. Dr. Waldrop has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapies.
Kevin M. Flanigan, MD, FAAN (Nationwide CHildrens Hospital)	Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Armatus. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. The institution of Dr. Flanigan has received research support from Ultragenyx . The institution of Dr. Flanigan has received research support from Solid Biosciences. The institution of Dr. Flanigan has received research support from Dyne Therapeutics. The institution of Dr. Flanigan has received research support from Avidity.
Jerry R. Mendell, MD, FAAN (The Research Institute at Nationwide Children's Hospital)	Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.
Linda P. Lowes, PT PhD	The institution of Ms. Lowes has received research support from Sarepta Therapeutics.

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