European Journal of Paediatric Neurology 
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This is an RSS file. You can use it to subscribe to this data in your favourite RSS reader or to display this data on your own website or blog.Validation of the Observer-Reported Communication Ability (ORCA) measure for individuals with Rett syndrome
CONCLUSIONS: This study provided initial support that the modified ORCA measure is an acceptable caregiver-reported measure of communication ability for females with RTT. Future work should include evaluation of longitudinal validity of the measure and its associations with clinician- and performance-based measures in diverse samples.PMID:37536121 | DOI:10.1016/j.ejpn.2023.07.007 (Source: European Journal of Paediatric Neurology)
Source: European Journal of Paediatric Neurology - August 3, 2023 Category: Neurology Authors: Bryce B Reeve Nicole Lucas Dandan Chen Molly McFatrich Harrison N Jones Kelly L Gordon Leslie Zapata Leiva Li Lin Monica Coenraads Jana von Hehn Randall L Carpenter Eric D Marsh Christina K Zigler Source Type: research
Health-related quality of life of children with spinal muscular atrophy in Sweden: A prospective cohort study in the era of disease-modifying therapy
In conclusion, we show that SMA has a detrimental impact on HRQoL that extends above and beyond somatic disability. Children with SMA type II experienced a dramatic increase in HRQoL over time, predominantly related to improvement in physical and social functioning. Our data helps quantify the patient burden of disease and adds to the rapidly expanding body of evidence of the effectiveness of recently approved disease-modifying therapies for SMA.PMID:37473536 | DOI:10.1016/j.ejpn.2023.06.008 (Source: European Journal of Paediatric Neurology)
Source: European Journal of Paediatric Neurology - July 20, 2023 Category: Neurology Authors: Erik Landfeldt Camilla Udo Malin L övgren Thomas Sejersen Ulrika Kreicbergs Source Type: research
Health-related quality of life of children with spinal muscular atrophy in Sweden: A prospective cohort study in the era of disease-modifying therapy
In conclusion, we show that SMA has a detrimental impact on HRQoL that extends above and beyond somatic disability. Children with SMA type II experienced a dramatic increase in HRQoL over time, predominantly related to improvement in physical and social functioning. Our data helps quantify the patient burden of disease and adds to the rapidly expanding body of evidence of the effectiveness of recently approved disease-modifying therapies for SMA.PMID:37473536 | DOI:10.1016/j.ejpn.2023.06.008 (Source: European Journal of Paediatric Neurology)
Source: European Journal of Paediatric Neurology - July 20, 2023 Category: Neurology Authors: Erik Landfeldt Camilla Udo Malin L övgren Thomas Sejersen Ulrika Kreicbergs Source Type: research
Health-related quality of life of children with spinal muscular atrophy in Sweden: A prospective cohort study in the era of disease-modifying therapy
In conclusion, we show that SMA has a detrimental impact on HRQoL that extends above and beyond somatic disability. Children with SMA type II experienced a dramatic increase in HRQoL over time, predominantly related to improvement in physical and social functioning. Our data helps quantify the patient burden of disease and adds to the rapidly expanding body of evidence of the effectiveness of recently approved disease-modifying therapies for SMA.PMID:37473536 | DOI:10.1016/j.ejpn.2023.06.008 (Source: European Journal of Paediatric Neurology)
Source: European Journal of Paediatric Neurology - July 20, 2023 Category: Neurology Authors: Erik Landfeldt Camilla Udo Malin L övgren Thomas Sejersen Ulrika Kreicbergs Source Type: research
Health-related quality of life of children with spinal muscular atrophy in Sweden: A prospective cohort study in the era of disease-modifying therapy
In conclusion, we show that SMA has a detrimental impact on HRQoL that extends above and beyond somatic disability. Children with SMA type II experienced a dramatic increase in HRQoL over time, predominantly related to improvement in physical and social functioning. Our data helps quantify the patient burden of disease and adds to the rapidly expanding body of evidence of the effectiveness of recently approved disease-modifying therapies for SMA.PMID:37473536 | DOI:10.1016/j.ejpn.2023.06.008 (Source: European Journal of Paediatric Neurology)
Source: European Journal of Paediatric Neurology - July 20, 2023 Category: Neurology Authors: Erik Landfeldt Camilla Udo Malin L övgren Thomas Sejersen Ulrika Kreicbergs Source Type: research
