Revised upper limb module for spinal muscular atrophy: Development of a new module

Elena S Mazzone; Anna Mayhew; Jacqueline Montes; Danielle Ramsey; Lavinia Fanelli; Sally Dunaway Young; Rachel Salazar; Roberto De Sanctis; Amy Pasternak; Allan Glanzman; Giorgia Coratti; Matthew Civitello; Nicola Forcina; Richard Gee; Tina Duong; Marika Pane; Mariacristina Scoto; Maria Carmela Pera; Sonia Messina; Gihan Tennekoon; John W Day; Basil T Darras; Darryl C De Vivo; Richard Finkel; Francesco Muntoni; Eugenio Mercuri

doi:10.1002/mus.25430

Revised upper limb module for spinal muscular atrophy: Development of a new module

Elena S Mazzone, Anna Mayhew, Jacqueline Montes, Danielle Ramsey, Lavinia Fanelli, Sally Dunaway Young, Rachel Salazar, Roberto De Sanctis, Amy Pasternak, Allan Glanzman, Giorgia Coratti, Matthew Civitello, Nicola Forcina, Richard Gee, Tina Duong, Marika Pane, Mariacristina Scoto, Maria Carmela Pera, Sonia Messina, Gihan TennekoonJohn W Day, Basil T Darras, Darryl C De Vivo, Richard Finkel, Francesco Muntoni, Eugenio Mercuri

School of Health and Social Work

Research output: Contribution to journal › Article › peer-review

62 Citations (Scopus)

Abstract

INTRODUCTION: There is a growing need for a robust clinical measure to assess upper limb motor function in spinal muscular atrophy (SMA), as the available scales lack sensitivity at the extremes of the clinical spectrum. We report the development of the Revised Upper Limb Module (RULM), an assessment specifically designed for upper limb function in SMA patients.

METHODS: An international panel with specific neuromuscular expertise performed a thorough review of scales currently available to assess upper limb function in SMA. This review facilitated a revision of the existing upper limb function scales to make a more robust clinical scale.

RESULTS: Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups.

CONCLUSIONS: The resulting RULM scale shows good reliability and validity, making it a suitable tool to assess upper extremity function in the SMA population for multi-center clinical research. Muscle Nerve, 2016.

Original language	English
Pages (from-to)	869-874
Number of pages	6
Journal	Muscle & Nerve
Volume	55
Issue number	6
Early online date	6 Feb 2017
DOIs	https://doi.org/10.1002/mus.25430
Publication status	Published - 1 Jun 2017

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Mazzone, E. S., Mayhew, A., Montes, J., Ramsey, D., Fanelli, L., Young, S. D., Salazar, R., De Sanctis, R., Pasternak, A., Glanzman, A., Coratti, G., Civitello, M., Forcina, N., Gee, R., Duong, T., Pane, M., Scoto, M., Pera, M. C., Messina, S., ... Mercuri, E. (2017). Revised upper limb module for spinal muscular atrophy: Development of a new module. Muscle & Nerve, 55(6), 869-874. https://doi.org/10.1002/mus.25430

@article{12649a762750414c9f54fe48dd2a4f77,

title = "Revised upper limb module for spinal muscular atrophy: Development of a new module",

abstract = "INTRODUCTION: There is a growing need for a robust clinical measure to assess upper limb motor function in spinal muscular atrophy (SMA), as the available scales lack sensitivity at the extremes of the clinical spectrum. We report the development of the Revised Upper Limb Module (RULM), an assessment specifically designed for upper limb function in SMA patients.METHODS: An international panel with specific neuromuscular expertise performed a thorough review of scales currently available to assess upper limb function in SMA. This review facilitated a revision of the existing upper limb function scales to make a more robust clinical scale.RESULTS: Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups.CONCLUSIONS: The resulting RULM scale shows good reliability and validity, making it a suitable tool to assess upper extremity function in the SMA population for multi-center clinical research. Muscle Nerve, 2016.",

author = "Mazzone, {Elena S} and Anna Mayhew and Jacqueline Montes and Danielle Ramsey and Lavinia Fanelli and Young, {Sally Dunaway} and Rachel Salazar and {De Sanctis}, Roberto and Amy Pasternak and Allan Glanzman and Giorgia Coratti and Matthew Civitello and Nicola Forcina and Richard Gee and Tina Duong and Marika Pane and Mariacristina Scoto and Pera, {Maria Carmela} and Sonia Messina and Gihan Tennekoon and Day, {John W} and Darras, {Basil T} and {De Vivo}, {Darryl C} and Richard Finkel and Francesco Muntoni and Eugenio Mercuri",

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Mazzone, ES, Mayhew, A, Montes, J, Ramsey, D, Fanelli, L, Young, SD, Salazar, R, De Sanctis, R, Pasternak, A, Glanzman, A, Coratti, G, Civitello, M, Forcina, N, Gee, R, Duong, T, Pane, M, Scoto, M, Pera, MC, Messina, S, Tennekoon, G, Day, JW, Darras, BT, De Vivo, DC, Finkel, R, Muntoni, F & Mercuri, E 2017, 'Revised upper limb module for spinal muscular atrophy: Development of a new module', Muscle & Nerve, vol. 55, no. 6, pp. 869-874. https://doi.org/10.1002/mus.25430

TY - JOUR

T1 - Revised upper limb module for spinal muscular atrophy

T2 - Development of a new module

AU - Mazzone, Elena S

AU - Mayhew, Anna

AU - Montes, Jacqueline

AU - Ramsey, Danielle

AU - Fanelli, Lavinia

AU - Young, Sally Dunaway

AU - Salazar, Rachel

AU - De Sanctis, Roberto

AU - Pasternak, Amy

AU - Glanzman, Allan

AU - Coratti, Giorgia

AU - Civitello, Matthew

AU - Forcina, Nicola

AU - Gee, Richard

AU - Duong, Tina

AU - Pane, Marika

AU - Scoto, Mariacristina

AU - Pera, Maria Carmela

AU - Messina, Sonia

AU - Tennekoon, Gihan

AU - Day, John W

AU - Darras, Basil T

AU - De Vivo, Darryl C

AU - Finkel, Richard

AU - Muntoni, Francesco

AU - Mercuri, Eugenio

PY - 2017/6/1

Y1 - 2017/6/1

N2 - INTRODUCTION: There is a growing need for a robust clinical measure to assess upper limb motor function in spinal muscular atrophy (SMA), as the available scales lack sensitivity at the extremes of the clinical spectrum. We report the development of the Revised Upper Limb Module (RULM), an assessment specifically designed for upper limb function in SMA patients.METHODS: An international panel with specific neuromuscular expertise performed a thorough review of scales currently available to assess upper limb function in SMA. This review facilitated a revision of the existing upper limb function scales to make a more robust clinical scale.RESULTS: Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups.CONCLUSIONS: The resulting RULM scale shows good reliability and validity, making it a suitable tool to assess upper extremity function in the SMA population for multi-center clinical research. Muscle Nerve, 2016.

AB - INTRODUCTION: There is a growing need for a robust clinical measure to assess upper limb motor function in spinal muscular atrophy (SMA), as the available scales lack sensitivity at the extremes of the clinical spectrum. We report the development of the Revised Upper Limb Module (RULM), an assessment specifically designed for upper limb function in SMA patients.METHODS: An international panel with specific neuromuscular expertise performed a thorough review of scales currently available to assess upper limb function in SMA. This review facilitated a revision of the existing upper limb function scales to make a more robust clinical scale.RESULTS: Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups.CONCLUSIONS: The resulting RULM scale shows good reliability and validity, making it a suitable tool to assess upper extremity function in the SMA population for multi-center clinical research. Muscle Nerve, 2016.

U2 - 10.1002/mus.25430

DO - 10.1002/mus.25430

M3 - Article

C2 - 27701745

SN - 1097-4598

VL - 55

SP - 869

EP - 874

JO - Muscle & Nerve

JF - Muscle & Nerve

IS - 6

ER -

Revised upper limb module for spinal muscular atrophy: Development of a new module

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