Self-reported functioning among patients with ultra-rare nemaline myopathy or a related disorder in Finland: a pilot study

Self-reported functioning among patients with ultra-rare nemaline myopathy or a related disorder in Finland: a pilot study

Abstract Background Nemaline myopathy (NM) and related disorders (NMr) form a heterogenous group of ultra-rare (1:50,000 live births or less) congenital muscle disorders. To elucidate the self-reported physical, psychological, and social functioning in the daily lives of adult persons with congenita...

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Bibliographic Details
Main Authors: Vilma-Lotta Lehtokari, Minna Similä, Marianne Tammepuu, Carina Wallgren-Pettersson, Sonja Strang-Karlsson, Sinikka Hiekkala
Format: Article
Language:English
Published: BMC 2023-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Congenital myopathies
Cross-sectional survey
International classification of functioning (ICF)
Self-reported functioning
Nemaline myopathy
Patient-Reported Outcomes Measurement Information System (PROMIS®)
Online Access:https://doi.org/10.1186/s13023-023-02973-2

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https://doi.org/10.1186/s13023-023-02973-2

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