ArticleRevista de neurologia2025
[Spinal Muscular Atrophy: The Reality of the Adult Patient in Spain].
Article in Revista de neurologia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundSpinal muscular atrophy (SMA) is a neurodegenerative disease in which there has been an increase in survival over the past decade due to proactive, multidisciplinary management and the emergence of disease-modifying therapies. Despite this, there are still several critical challenges that significantly impact the quality of life of individuals with SMA individuals. Given the need to better understand the reality of this disease, Fundación de Atrofia Muscular Espinal (FundAME) created a patient registry (RegistrAME).
methodsRegistrAME is a longitudinal prospective study that collects clinical data and patient-reported outcomes.
resultsThe registry included 336 individuals, 51.8% of whom were over 16 years old. Most adult subpopulations were classed as type 2 (49.4%) or type 3 (44.8%). Regarding functional status, 19% walked (39.4% used wheelchairs), 46.6% sat without support (84% needed help to sit), and 34.5% were non-sitters. Furthermore, 24.7% reported having no useful function in their hands or not being able to reach their mouths with their hands. Our study indicates that the adult SMA population is progressively accessing disease-modifier treatments. However, 21.8% of the population is still untreated.
conclusionsThese results provide evidence of a progressive impairment and increased sensitivity to treatment discontinuations in the SMA adult population, along with delayed or reduced access to commercialised SMA drugs and clinical trials. A more rigorous evaluation of the disease's impact and treatment benefits in the adult SMA population is necessary.
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