title: Revised upper limb module for spinal muscular atrophy: Development of a new module.
abstract: 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.
  
  
  
  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.
  
  
  
  Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups.
  
  
  
  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 55: 869-874, 2017.
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Elena S
authors.initials: ES
authors.lastname: Mazzone
authors.affiliation: Institute of Genetic Medicine, Newcastle University, Newcastle, United Kingdom.
authors.forename: Anna
authors.initials: A
authors.lastname: Mayhew
authors.affiliation: Department of Neurology, Columbia University Medical Center, New York, USA.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Montes
authors.affiliation: Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
authors.forename: Danielle
authors.initials: D
authors.lastname: Ramsey
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Lavinia
authors.initials: L
authors.lastname: Fanelli
authors.affiliation: Department of Neurology, Columbia University Medical Center, New York, USA.
authors.forename: Sally Dunaway
authors.initials: SD
authors.lastname: Young
authors.affiliation: Department of Neurology, Columbia University Medical Center, New York, USA.
authors.forename: Rachel
authors.initials: R
authors.lastname: Salazar
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Amy
authors.initials: A
authors.lastname: Pasternak
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia and Departments of Neurology and Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA.
authors.forename: Allan
authors.initials: A
authors.lastname: Glanzman
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Giorgia
authors.initials: G
authors.lastname: Coratti
authors.affiliation: Division of Neurology, Nemours Children's Hospital, Orlando, Florida, USA.
authors.forename: Matthew
authors.initials: M
authors.lastname: Civitello
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Nicola
authors.initials: N
authors.lastname: Forcina
authors.affiliation: Department of Neurology, Stanford University, Stanford, California, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Gee
authors.affiliation: Department of Neurology, Stanford University, Stanford, California, USA.
authors.forename: Tina
authors.initials: T
authors.lastname: Duong
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.affiliation: Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
authors.forename: Mariacristina
authors.initials: M
authors.lastname: Scoto
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Maria Carmela
authors.initials: MC
authors.lastname: Pera
authors.affiliation: Department of Clinical and Experimental Medicine and Nemo Sud Clinical Centre, University of Messina, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia and Departments of Neurology and Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA.
authors.forename: Gihan
authors.initials: G
authors.lastname: Tennekoon
authors.affiliation: Department of Neurology, Stanford University, Stanford, California, USA.
authors.forename: John W
authors.initials: JW
authors.lastname: Day
authors.affiliation: Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Basil T
authors.initials: BT
authors.lastname: Darras
authors.affiliation: Department of Neurology, Columbia University Medical Center, New York, USA.
authors.forename: Darryl C
authors.initials: DC
authors.lastname: De Vivo
authors.affiliation: Division of Neurology, Nemours Children's Hospital, Orlando, Florida, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Finkel
authors.affiliation: Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D004185:Disability Evaluation; D005260:Female; D006801:Humans; D007391:International Cooperation; D007558:Italy; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000473:pathology*; D011594:Psychometrics; D015203:Reproducibility of Results; D012720:Severity of Illness Index; D006113:United Kingdom; D014481:United States; D034941:Upper Extremity / Q000503:physiopathology*
keywords: Hammersmith functional motor scale; Rasch analysis; functional assessment; outcome measures; spinal muscular atrophy; upper limb function