PMID- 29139300
OWN - NLM
STAT- MEDLINE
DCOM- 20190919
LR  - 20191210
IS  - 2042-6313 (Electronic)
IS  - 2042-6305 (Linking)
VI  - 7
IP  - 4
DP  - 2018 Apr
TI  - Development and preliminary evidence of the psychometric properties of the GNE 
      myopathy functional activity scale.
PG  - 381-395
LID - 10.2217/cer-2017-0062 [doi]
AB  - AIM: GNE myopathy, a rare, severe, progressive myopathy, presents with lower 
      extremity distal muscle weakness. The GNE myopathy functional activity scale 
      (GNEM-FAS) evaluates the impact of GNE myopathy on functioning in adults. This 
      paper presents the psychometric validation of the GNEM-FAS. PATIENTS & METHODS: 
      Validation of the GNEM-FAS was performed using data from a randomized, 
      double-blind, placebo-controlled Phase-II study (n = 46). RESULTS: Domain score 
      distributions were acceptable. Moderate inter-item correlations (typical range, 
      0.40-0.70), strong item convergent and discriminant validity and high internal 
      consistency reliability (alpha = 0.88-0.92) supported the instrument structure. 
      Test-retest reliability was strong (ICC range: 0.87-0.95). Scale scores 
      distinguished among subjects with differing disease severity (p < 0.05). 
      CONCLUSION: This study provides preliminary evidence of the GNEM-FAS as a valid, 
      reliable assessment.
FAU - Mayhew, Jill
AU  - Mayhew J
AD  - Ultragenyx Pharmaceutical, 60 Leveroni Court, Novato, CA 94949, USA.
FAU - Bonner, Nicola
AU  - Bonner N
AD  - Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
FAU - Arbuckle, Rob
AU  - Arbuckle R
AD  - Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
FAU - Turnbull, Alice
AU  - Turnbull A
AD  - Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
FAU - Bowden, Alexandra
AU  - Bowden A
AD  - Ultragenyx Pharmaceutical, 60 Leveroni Court, Novato, CA 94949, USA.
FAU - Skrinar, Alison
AU  - Skrinar A
AD  - Ultragenyx Pharmaceutical, 60 Leveroni Court, Novato, CA 94949, USA.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Validation Study
DEP - 20171115
PL  - England
TA  - J Comp Eff Res
JT  - Journal of comparative effectiveness research
JID - 101577308
SB  - IM
MH  - Adult
MH  - Aged
MH  - Clinical Trials, Phase II as Topic
MH  - *Disability Evaluation
MH  - Distal Myopathies/genetics/*physiopathology
MH  - Double-Blind Method
MH  - Female
MH  - Humans
MH  - Interviews as Topic
MH  - Male
MH  - Middle Aged
MH  - Psychometrics
MH  - Qualitative Research
MH  - Randomized Controlled Trials as Topic
MH  - Reproducibility of Results
OTO - NOTNLM
OT  - GNE myopathy
OT  - functional activity
OT  - psychometric validation
EDAT- 2017/11/16 06:00
MHDA- 2019/09/20 06:00
CRDT- 2017/11/16 06:00
PHST- 2017/11/16 06:00 [pubmed]
PHST- 2019/09/20 06:00 [medline]
PHST- 2017/11/16 06:00 [entrez]
AID - 10.2217/cer-2017-0062 [doi]
PST - ppublish
SO  - J Comp Eff Res. 2018 Apr;7(4):381-395. doi: 10.2217/cer-2017-0062. Epub 2017 Nov 
      15.