Accepted for Publication: April 16, 2013.
Published Online: June 24, 2013. doi:10.1001/jamaneurol.2013.3178.
Study concept and design: Lewis, McDermott, Herrmann, Hoke, Wu, and Shy.
Acquisition of data: Lewis, Herrmann, Hoke, Clawson, Siskind, Feely, Miller,
Smith, Luebbe, Wu, and Shy.
Analysis and interpretation of data: Lewis, McDermott, Herrmann, Hoke, Barohn,
Wu, and Shy.
Drafting of the manuscript: Lewis, Hoke, Clawson, Smith, Wu, and Shy.
Critical revision of the manuscript for important intellectual content: Lewis,
McDermott, Herrmann, Hoke, Siskind, Feely, Miller, Barohn, Luebbe, Wu, and Shy.
Statistical analysis: McDermott and Wu.
Obtained funding: Hoke and Shy.
Administrative, technical, and material support: Herrmann, Hoke, Siskind, Feely,
Miller, Smith, Luebbe, Wu, and Shy.
Study supervision: Lewis, Herrmann, Hoke, Siskind, Barohn, Smith, and Shy.
Conflict of Interest Disclosures: Dr Lewis has
consulted for Baxter, CSL Behring, AxelaCare, Novartis, and Bristol-Myers Squibb. Dr Shy has
received grant support from the National Institute of Neurological Disorders and Stroke, Muscular
Dystrophy Association, and Charcot-Marie-Tooth Association.
Funding/Support: This work was supported by the
Muscular Dystrophy Association and the Charcot-Marie-Tooth Association. Wayne State University, the University
of Rochester, and Johns Hopkins University were members of the Inherited Neuropathy Consortium of
the Rare Disease Clinical Research Network supported by the National
Institute of Neurological Disorders and Stroke and Office
of Rare Diseases.
Muscle Study Group Members: Richard A. Lewis,
MD; Michael P. McDermott, PhD; David N. Herrmann, MD; Ahmet Hoke, MD, PhD; Lora L. Clawson, MSN,
CRNP; Carly Siskind, MS; Shawna M. E. Feely, MS; Lindsey J. Miller, MS; Richard J. Barohn, MD;
Patricia Smith, BS; Elizabeth Luebbe, MS; Xingyao Wu, MD, PhD; Michael E. Shy, MD; Rabi Tawil, MD;
Berch Griggs, MD; Colleen Donlin Smith, MA, CCRC; William Martens; Arthur Watts; Steve Bean, PharmD;
Eileen Eastwood; Nuran Dilek, MS; and Carrie Irvine.
Additional Information: The full trial protocol is available from Dr Shy
Additional Contributions: We thank all the subjects who participated in the trial.
Lisa Rowe, BA, worked in coordinating subject visits, Karen M. Krajewski, MS, helped in designing
the protocol, Richard Kimball, RN, PhD, helped with subject recruitment and evaluations, and Arthur
Watts, BS, helped with the statistical analysis.