PMID- 23681930
OWN - NLM
STAT- MEDLINE
DCOM- 20131111
LR  - 20220408
IS  - 1097-4598 (Electronic)
IS  - 0148-639X (Print)
IS  - 0148-639X (Linking)
VI  - 48
IP  - 3
DP  - 2013 Sep
TI  - The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: 
      longitudinal natural history observations over 48 weeks from a multicenter study.
PG  - 343-56
LID - 10.1002/mus.23902 [doi]
AB  - INTRODUCTION: Duchenne muscular dystrophy (DMD) subjects >/=5 years with nonsense 
      mutations were followed for 48 weeks in a multicenter, randomized, double-blind, 
      placebo-controlled trial of ataluren. Placebo arm data (N = 57) provided insight 
      into the natural history of the 6-minute walk test (6MWT) and other endpoints. 
      METHODS: Evaluations performed every 6 weeks included the 6-minute walk distance 
      (6MWD), timed function tests (TFTs), and quantitative strength using hand-held 
      myometry. RESULTS: Baseline age (>/=7 years), 6MWD, and selected TFT performance 
      are strong predictors of decline in ambulation (Delta6MWD) and time to 10% worsening 
      in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional 
      decline, and loss of ambulation was only seen in those with baseline 6MWD <325 
      meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. 
      CONCLUSION: Findings confirm the clinical meaningfulness of the 6MWD as the most 
      accepted primary clinical endpoint in ambulatory DMD trials.
CI  - Copyright (c) 2013 Wiley Periodicals, Inc.
FAU - McDonald, Craig M
AU  - McDonald CM
AD  - Department of Physical Medicine and Rehabilitation, University of California 
      Davis School of Medicine, Davis, California, 95817, USA.
FAU - Henricson, Erik K
AU  - Henricson EK
FAU - Abresch, R Ted
AU  - Abresch RT
FAU - Florence, Julaine M
AU  - Florence JM
FAU - Eagle, Michelle
AU  - Eagle M
FAU - Gappmaier, Eduard
AU  - Gappmaier E
FAU - Glanzman, Allan M
AU  - Glanzman AM
CN  - PTC124-GD-007-DMD Study Group
FAU - Spiegel, Robert
AU  - Spiegel R
FAU - Barth, Jay
AU  - Barth J
FAU - Elfring, Gary
AU  - Elfring G
FAU - Reha, Allen
AU  - Reha A
FAU - Peltz, Stuart
AU  - Peltz S
LA  - eng
GR  - MR/K000608/1/MRC_/Medical Research Council/United Kingdom
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
DEP - 20130626
PL  - United States
TA  - Muscle Nerve
JT  - Muscle & nerve
JID - 7803146
RN  - 0 (Glucocorticoids)
RN  - 0 (Oxadiazoles)
RN  - K16AME9I3V (ataluren)
SB  - IM
CIN - Muscle Nerve. 2013 Sep;48(3):315-7. PMID: 24038058
MH  - Adolescent
MH  - Child
MH  - Child, Preschool
MH  - Double-Blind Method
MH  - Electromyography
MH  - *Exercise Test
MH  - Glucocorticoids/therapeutic use
MH  - Hand Strength/physiology
MH  - Humans
MH  - Longitudinal Studies
MH  - Male
MH  - Muscular Dystrophy, Duchenne/drug therapy/*physiopathology
MH  - Observation
MH  - *Outcome Assessment, Health Care
MH  - Oxadiazoles/therapeutic use
MH  - Predictive Value of Tests
MH  - Time Factors
MH  - Walking/*physiology
PMC - PMC3824082
OTO - NOTNLM
OT  - 6-minute walk test
OT  - Duchenne muscular dystrophy
OT  - ambulation
OT  - dystrophinopathy
OT  - myometry
OT  - natural history
OT  - prediction of loss of function
OT  - timed function tests
FIR - Ryan, Monique
IR  - Ryan M
FIR - Jones, Kristi
IR  - Jones K
FIR - Goemans, Nathalie
IR  - Goemans N
FIR - Campbell, Craig
IR  - Campbell C
FIR - Mah, Jean
IR  - Mah J
FIR - Selby, Kathryn
IR  - Selby K
FIR - Voit, Thomas
IR  - Voit T
FIR - Chabrol, Brigitte
IR  - Chabrol B
FIR - Pereon, Yann
IR  - Pereon Y
FIR - Schara, Ulrike
IR  - Schara U
FIR - Kirschner, Janberndt
IR  - Kirschner J
FIR - Bertini, Enrico
IR  - Bertini E
FIR - Mercuri, Eugenio
IR  - Mercuri E
FIR - Comi, Giacomo
IR  - Comi G
FIR - Nevo, Yoram
IR  - Nevo Y
FIR - Vilchez, Juan
IR  - Vilchez J
FIR - Colomer, Jaume
IR  - Colomer J
FIR - Children, Sylvia
IR  - Children S
FIR - Tulinius, Mar
IR  - Tulinius M
FIR - Sejersen, Thomas
IR  - Sejersen T
FIR - Bushby, Katherine
IR  - Bushby K
FIR - Muntoni, Francesco
IR  - Muntoni F
FIR - Quinlivan, Rosaline Christina Mary
IR  - Quinlivan RC
FIR - Wong, Brenda
IR  - Wong B
FIR - Finkel, Richard S
IR  - Finkel RS
FIR - Sampson, Jacinda B
IR  - Sampson JB
FIR - Flanigan, Kevin M
IR  - Flanigan KM
FIR - Butterfield, Russell
IR  - Butterfield R
FIR - Day, John W
IR  - Day JW
FIR - Mathews, Katherine
IR  - Mathews K
FIR - Darras, Basil T
IR  - Darras BT
FIR - Apkon, Susan D
IR  - Apkon SD
FIR - Parsons, Julie
IR  - Parsons J
FIR - Barohn, Richard
IR  - Barohn R
FIR - Connolly, Anne
IR  - Connolly A
FIR - Iannaccone, Susan
IR  - Iannaccone S
FIR - Sproule, Douglas M
IR  - Sproule DM
FIR - Kaufman, Petra
IR  - Kaufman P
FIR - Han, Jay J
IR  - Han JJ
FIR - Joyce, Nanette C
IR  - Joyce NC
FIR - Renfroe, J Ben
IR  - Renfroe J
FIR - Russman, Barry S
IR  - Russman BS
FIR - Burns-Wechsler, Stephanie
IR  - Burns-Wechsler S
FIR - Moore, Steven A
IR  - Moore SA
FIR - Sweeney, H Lee
IR  - Sweeney HL
FIR - Coleman, Kim
IR  - Coleman K
EDAT- 2013/05/18 06:00
MHDA- 2013/11/12 06:00
CRDT- 2013/05/18 06:00
PHST- 2013/05/07 00:00 [accepted]
PHST- 2013/05/18 06:00 [entrez]
PHST- 2013/05/18 06:00 [pubmed]
PHST- 2013/11/12 06:00 [medline]
AID - 10.1002/mus.23902 [doi]
PST - ppublish
SO  - Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26.