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Characteristics and outcomes of patients with LAM receiving sirolimus in France based on real-life data.
Front Med (Lausanne)
January 2025
Department of Respiratory Medicine, Reference Centre for Rare Pulmonary Diseases, APHP, Hôpital Avicenne, INSERM U 1272, Université Sorbonne Paris-Nord, Bobigny, France.
LAM is a rare multi-cystic lung disease for which treatment with sirolimus is indicated in cases of moderate or severe lung disease or declining lung function. The aim of this study was to describe patients treated with sirolimus for LAM and their outcomes. This retrospective observational study was based on data from the French national health insurance data system (SNDS).
View Article and Find Full Text PDFPsychosocial moderators of the association between pain intensity and physical function in people with systemic sclerosis.
J Scleroderma Relat Disord
January 2025
Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, MI, USA.
Background: Pain is a prevalent symptom of systemic sclerosis. While previous studies have demonstrated a correlation between higher pain intensity and lower physical function in individuals with systemic sclerosis, the potentially moderating effect of psychosocial factors on the association has yet to be explored.
Methods: This cross-sectional study used data from a fatigue self-management trial for adults with systemic sclerosis.
Use of Lung Volume Recruitment Technique in Patients With Chronic Respiratory Disease Among Brazilian Health Professionals.
Pulm Med
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Post Graduation Department, Escola Superior de Ciências da Saúde (ESCS), Brasilia, Distrito Federal, Brazil.
Lung volume recruitment (LVR) is a stacked-breath assisted inflation technique in which consecutive insufflations are delivered, without exhaling in between, until the maximum tolerable inflation capacity is reached. Although LVR is recommended in some neuromuscular disease guidelines, there is little information detailing when and how allied health professionals (AHPs) prescribe LVR. This study is aimed at describing the use of LVR in practice across Brazil.
View Article and Find Full Text PDFElectrophysiological Abnormalities in Finger Extension Weakness and DOwnbeat Nystagmus Motor Neuron Disease: Three New Patients and Review of the Literature.
Muscle Nerve
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Service ENMG et de Pathologies Neuromusculaires, Centre de référence Des Maladies Neuromusculaires PACA-Réunion-Rhône Alpes, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, Bron, France.
Introduction/aims: Finger Extension Weakness and DOwnbeat Nystagmus Motor Neuron Disease (FEWDON-MND) is characterized by motor weakness predominantly affecting finger extension, accompanied by downbeat nystagmus. To date, only 11 patients have been reported. The present study adds a further three and aims to provide a more detailed description of the electrodiagnostic features of these patients.
View Article and Find Full Text PDFAdvances in RNA therapy for the treatment of autoimmune diseases.
Autoimmun Rev
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The Department of Dermatology, Xiangya Hospital, Central South University, Changsha, Hunan, China; National Clinical Research Center for Geriatric Disorders, Xiangya Hospital, Changsha, Hunan, China; Hunan Key Laboratory of Skin Cancer and Psoriasis, Xiangya Hospital, Changsha, Hunan, China; Hunan Engineering Research Center of Skin Health and Disease, Xiangya Hospital, Changsha, Hunan, China. Electronic address:
Autoimmune diseases (ADs) are a group of complex, chronic conditions characterized by disturbance of immune tolerance, with examples including systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, and psoriasis. These diseases have unclear pathogenesis, and traditional therapeutic approaches remain limited. However, advances in high-throughput histology technology and scientific discoveries have led to the identification of various pathogenic factors contributing to ADs.
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