The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy

Neuromuscular Disorders : NMD
Leslie NelsonAmSMART Group

Abstract

Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure is a measure of motor function. We showed previously that the Gross Motor Function Measure is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P<0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy.

References

Oct 26, 2001·Journal of the Neurological Sciences·R G MillerUNKNOWN SMA Study Group
Jun 29, 2002·Journal of Child Neurology·Aviva Fattal-ValevskiShlomo Hayek
Aug 7, 2002·Developmental Medicine and Child Neurology·Melissa L McCarthyNancy A Hadley-Miller
Aug 28, 2002·Neurology·M KinaliF Muntoni
Sep 12, 2002·Archives of Neurology·Susan T Iannaccone, UNKNOWN American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group
Aug 20, 2003·Archives of Neurology·Susan T IannacconeUNKNOWN American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group
Sep 25, 2003·Muscle & Nerve·Hafedh HaddadJudith Melki
Nov 5, 2003·Annals of Neurology·Charlotte J SumnerKenneth H Fischbeck
Jan 22, 2004·Neuromuscular Disorders : NMD·Eugenio MercuriChristina Brahe

❮ Previous
Next ❯

Citations

Feb 16, 2011·Archives of Neurology·Petra KaufmannUNKNOWN Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy
Apr 23, 2013·Archives of Physical Medicine and Rehabilitation·Capucine de LattreUNKNOWN MFM-20 Study Group
Apr 2, 2013·Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association·Kristin J KrosschellUNKNOWN Project Cure SMA Investigators Network
Jul 1, 2010·Journal of Child Neurology·Susan Sienko ThomasMichael D Sussman
Oct 19, 2012·Neurology·Petra KaufmannUNKNOWN Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR)
Apr 9, 2013·PloS One·Dione T KobayashiKaren S Chen
Apr 8, 2015·Muscle & Nerve·Jacqueline MontesUNKNOWN Pediatric Neuromuscular Clinical Research Network, Muscle Study Group, SMA Europe
Apr 21, 2012·Lancet Neurology·Eugenio MercuriSusan T Iannaccone
Dec 27, 2011·Pediatric Neurology·Jennifer A MarkowitzBasil T Darras
Mar 23, 2011·Neuromuscular Disorders : NMD·Elena MazzoneEugenio Mercuri
Jul 16, 2008·Physical Medicine and Rehabilitation Clinics of North America·Jay J Han, Craig M McDonald
Jul 4, 2008·Neuromuscular Disorders : NMD·Carole BérardChristine Payan
Jul 31, 2007·Neuromuscular Disorders : NMD·Jessica M O'HagenDarryl C De Vivo
Apr 24, 2008·Developmental Medicine and Child Neurology·Eugenio MercuriEnrico Bertini
Jun 24, 2011·Muscle & Nerve·Kristin J KrosschellUNKNOWN Project Cure SMA
Jul 31, 2013·Muscle & Nerve·Peter B KangUNKNOWN Muscle Study Group and the Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy
Jul 10, 2013·Muscle & Nerve·Stefan J CanoUNKNOWN International Coordinating Committee for SMA Clinical Trials Rasch Task Force
Sep 28, 2010·Physical & Occupational Therapy in Pediatrics·Yasser Salem, Stacy Jaffee Gropack
May 17, 2014·Molecular Genetics and Metabolism·Tatsuya FujiiTomohiro Kumada
Feb 6, 2013·Archives of Physical Medicine and Rehabilitation·Carole VuillerotUNKNOWN MFM Spinal Muscular Atrophy Study Group
Nov 8, 2016·Neuromuscular Disorders : NMD·Takatoshi SatoKeiko Ishigaki
Sep 5, 2014·Journal of Child Neurology·Lisa A RothDouglas M Sproule
Dec 14, 2011·Journal of Child Neurology·Sally DunawayDarryl C De Vivo
Sep 24, 2011·Journal of Child Neurology·Allan M GlanzmanUNKNOWN Muscle Study Group (MSG)
Sep 1, 2007·Journal of Child Neurology·Ching H WangUNKNOWN Participants of the International Conference on SMA Standard of Care
Oct 12, 2010·Journal of Child Neurology·Douglas M SproulePetra Kaufmann
Jun 11, 2009·Journal of Child Neurology·Jacqueline MontesPetra Kaufmann
May 2, 2013·Therapeutic Advances in Neurological Disorders·Wendy K M Liew, Peter B Kang
Jun 21, 2018·Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association·Rachel SalazarDarryl C De Vivo
Jul 25, 2013·Annals of Human Genetics·Dian K NurputraHisahide Nishio
Dec 14, 2019·BMJ Supportive & Palliative Care·Colleen PawliukHarold Hal Siden
Jul 7, 2018·Child Neurology Open·Christina StarkEckhard Schoenau
May 16, 2021·Orphanet Journal of Rare Diseases·J SchaefersH H Huidekoper
May 18, 2021·Child Neurology Open·Katarzyna PierzchlewiczKatarzyna Kotulska
Jun 11, 2021·Orphanet Journal of Rare Diseases·Jennifer M BainSylvie Goldman
Oct 3, 2007·Pediatrics·Michaela Linder-LuchtUNKNOWN Gross Motor Function Measure-Traumatic Brain Injury Study Group
Dec 28, 2021·Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association·Anna Te Velde, Catherine Morgan

❮ Previous
Next ❯

Related Concepts

Related Feeds

Cajal Bodies & Gems

Cajal bodies or coiled bodies are dense foci of coilin protein. Gemini of Cajal bodies, or gems, are microscopically similar to Cajal bodies. It is believed that Cajal bodies play important roles in RNA processing while gems assist the Cajal bodies. Find the latest research on Cajal bodies and gems here.

© 2022 Meta ULC. All rights reserved