University of Hertfordshire

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Original languageEnglish
PagesS131
Number of pages1
DOIs
Publication statusPublished - Oct 2019
Event24th International Annual Congress of the World Muscle Society - Copenhagen, Denmark
Duration: 1 Oct 20195 Oct 2019
https://archive.worldmusclesociety.org/www.wms2019.com/

Conference

Conference24th International Annual Congress of the World Muscle Society
Abbreviated titleWMS 2019
CountryDenmark
CityCopenhagen
Period1/10/195/10/19
Internet address

Abstract

The Revised Hammersmith Scale (RHS) was developed by an international panel of experts to extend the range of functional abilities currently captured by the original Hammersmith Functional Scale Expanded (HFMSE), capturing the functional abilities of very weak Spinal Muscular Atrophy (SMA) type 2 patients through to very strong patients with SMA type 3. The scale consists of 36 items (total score 69), including timed tests and it is completed alongside the World Health Organization (WHO) motor milestones, thus enabling greater description of SMA disease trajectories. The RHS has shown to be a psychometrically sound and versatile clinical outcome assessment to test the broad range of physical abilities of patients with type 2 and 3 SMA and ongoing analysis is reviewing the relationship of the RHS with the HFMSE and Revised Upper Limb Module (RULM). The aim of this study is to further understand the longitudinal trajectories of motor function in a large international cohort (SMA REACH UK, PNCRN USA and Italian Telethon) of untreated patients with type 2 and 3 SMA using the RHS. To date more than 650 longitudinal assessments ranging from 6 to 24 months, in 180 patients aged between 1.2 years to 63.7 years, have been completed using the RHS. Out of 180 patients 103 had type 2 SMA and 77 had type 3 SMA. This ongoing study will provide a new insight into the natural history of untreated patients with SMA type 2 and 3 using the RHS, which is particularly relevant given the rapidly changing treatment landscape for SMA.

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