Results 71 to 80 of about 39,956 (308)
Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review
ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care.
Amin Mehrabian +9 more
wiley +1 more source
Variably Protease‐Sensitive Prionopathy: Two New Cases With Motor Neuron‐Dementia Syndrome
ABSTRACT We describe two patients with variably protease‐sensitive prionopathy (VPSPr) who developed progressive upper motor neuron symptoms, insomnia, behavioral and cognitive decline, compatible with primary lateral sclerosis associated with frontotemporal dementia (FTD).
María Elena Erro +10 more
wiley +1 more source
Whole‐Body Pattern of Muscle Degeneration and Progression in Sarcoglycanopathies
ABSTRACT Objective To characterize whole‐body intramuscular fat distribution pattern in patients with sarcoglycanopathies and explore correlations with disease severity, duration and age at onset. Methods Retrospective, cross‐sectional, multicentric study enrolling patients with variants in one of the four sarcoglycan genes who underwent whole‐body ...
Laura Costa‐Comellas +39 more
wiley +1 more source
THE EUROPEAN PARLIAMENT BEFORE THE 2014-2019 PARLIAMENTARY TERM IN THE LIGHT OF INNOVATIONS AND CHANGES INTRODUCED BY THE LISBON TREATY [PDF]
Viewed through the prism of its historical evolution, the European Parliament has seen an original evolutionary process during which underwent a series of successive modifications of its competences and composition, at the same time with increasing of ...
Dan VĂTĂMAN
doaj
Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points
ABSTRACT Objective Spinocerebellar ataxia type 3 (SCA3) is a genetically defined ataxia. The Scale for Assessment and Rating of Ataxia (SARA) is a clinician‐reported outcome that measures ataxia severity at a single time point. In its standard application, SARA fails to capture short‐term fluctuations, limiting its sensitivity in trials.
Marcus Grobe‐Einsler +20 more
wiley +1 more source
Refugee-led humanitarianism in Lebanon’s Shatila camp
Refugee-led humanitarian initiatives by ‘established’ Palestinian refugees in response to the arrival of ‘new’ displaced Syrians to Shatila camp raise key questions about the limitations of the humanitarian system and representations of refugees as ...
Hind Sharif
doaj
European Parliament adopts report on medicines shortages. [PDF]
Kohl S.
europepmc +1 more source
Objective The aim of this systematic review was to synthesize the economic impact of rheumatoid arthritis (RA) on households, health systems, and society in low‐ and middle‐income countries (LMICs). Methods Electronic databases such as PubMed, Web of Science, and CINAHL were searched using keywords related to RA and cost of illness.
Tadesse Gebrye +6 more
wiley +1 more source

