Abstract Details

Title Sensitive Assessment of Duchenne Muscular Dystrophy Progression with a Dedicated Electrical Impedance Myography Device

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S23 - Genetic Muscle Disorders (3:41 PM-3:52 PM)

Poster/Presentation
Number 002

Background EIM has been shown to be sensitive to DMD progression in younger boys. Whether a simple-to-use, dedicated device (the mView^TM, Myolex, Inc) provides similar results in both ambulatory and non-ambulatory boys is unknown.

Objective To determine the capability of a dedicated electrical impedance myography (EIM) system to serve as an assessment tool in Duchenne muscular dystrophy (DMD).

Design/Methods In this longitudinal study of DMD and healthy boys, participants were followed up to 1 year and assessed with the mView^TM system, the 6-minute walk test (6MWT), the North Star Ambulatory Assessment (NSAA), and handheld dynamometry (HHD).

Results The ambulatory group included 29 DMD and 44 healthy boys (mean ages±SD) (8.29±1.89 and 8.18±1.83 years, respectively) and the non-ambulatory group included 15 DMD and 13 healthy boys (14.6±1.33 and 14.4±1.08 years, respectively). In ambulatory DMD boys, EIM 100 kHz resistance values showed significant differences over both the 6-month and 1-year time periods compared to the healthy boys, particularly in tibialis anterior and quadriceps, giving an estimated effect size of 0.97 at 6 months, performing considerably better than the 6MWT (effect size 0.25) and the NSAA (effect size 0.44). Based on these EIM effect sizes, fewer than 100 DMD patients/arm would be needed for a 6-month trial assuming a 25% treatment effect. In non-ambulatory children, the 100 kHz resistance showed comparable changes in upper limb muscles, giving an estimated effect size of 1.9 at 12 months, with similar sample size estimates. Longitudinal EIM changes correlated with change in HHD and NSAA.

Conclusions This study supports that EIM measurements obtained with the mView device are sensitive to DMD progression in both ambulatory and non-ambulatory boys. Given the technology’s ease of use and acceptance by children of all ages, it should be considered for use as an outcome measure in future clinical therapeutic trials in DMD.

Authors/Disclosures
Seward B. Rutkove, MD, FAAN PRESENTER	Dr. Rutkove has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Myolex, Inc. Dr. Rutkove has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for American Academy of Neuorlogy . The institution of Dr. Rutkove has received research support from NIH. The institution of Dr. Rutkove has received research support from NASA. The institution of Dr. Rutkove has received research support from Blavatnik Family Foundation. Dr. Rutkove has received intellectual property interests from a discovery or technology relating to health care. Dr. Rutkove has received intellectual property interests from a discovery or technology relating to health care. Dr. Rutkove has received publishing royalties from a publication relating to health care. Dr. Rutkove has received publishing royalties from a publication relating to health care. Dr. Rutkove has received personal compensation in the range of $500-$4,999 for serving as a Grant Reviewer with NIH.
Basil T. Darras, MD (Children'S Hosp Boston Harvard Med School)	The institution of Dr. Darras has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Darras has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. The institution of Dr. Darras has received research support from National Institutes of Health/National Institute of Neurological Disorders and Stroke,. The institution of Dr. Darras has received research support from Slaney Family Fund for SMA. The institution of Dr. Darras has received research support from Spinal Muscular Atrophy Foundation. The institution of Dr. Darras has received research support from CureSMA. The institution of Dr. Darras has received research support from Working on Walking Fund . The institution of Dr. Darras has received research support from CHERISH, CS2/CS12 . The institution of Dr. Darras has received research support from Biogen for CS11. The institution of Dr. Darras has received research support from AveXis. The institution of Dr. Darras has received research support from Sarepta Pharmaceuticals. The institution of Dr. Darras has received research support from PTC Therapeutics. The institution of Dr. Darras has received research support from Roche. The institution of Dr. Darras has received research support from Santhera. The institution of Dr. Darras has received research support from Scholar Rock. The institution of Dr. Darras has received research support from Fibrogen. The institution of Dr. Darras has received research support from Summit. Dr. Darras has received publishing royalties from a publication relating to health care. Dr. Darras has received publishing royalties from a publication relating to health care.
Michele Yang, MD (Children'S Hospital Colorado)	The institution of Dr. Yang has received research support from Janssen. The institution of Dr. Yang has received research support from Edgewise. The institution of Dr. Yang has received research support from ReveraGen.
Brenda L. Wong, MD (University of Massachusetts Medical School)	No disclosure on file
	No disclosure on file
	No disclosure on file
Melanie Leitner, PhD (Accelerating NeuroVentures, LLC)	Dr. Leitner has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Prilenia. Dr. Leitner has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for ALS Investment Fund. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fundamental Pharma. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Caraway. Dr. Leitner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Affinia Therapeutics. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jannsen. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Tolerion/Alpha-5 Integrin. Dr. Leitner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Verge Genomics. Dr. Leitner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Tranquis. Dr. Leitner has stock in Bristol Myers Squibb. Dr. Leitner has stock in Editas. Dr. Leitner has stock in Provention Bio. Dr. Leitner has stock in Sanofi. Dr. Leitner has stock in Vertex. Dr. Leitner has stock in GSK. Dr. Leitner has stock in Eledon. Dr. Leitner has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant with ALS Finding a Cure. Dr. Leitner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant with Charley's Fund.
	No disclosure on file
Craig M. Zaidman, MD (Washington University in St Louis)	Dr. Zaidman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for sarepta. The institution of Dr. Zaidman has received research support from Washington University in St Louis. The institution of Dr. Zaidman has received research support from Novartis.

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