Omura Jaclyn, Stratton Anne, Fournier Christina, Howard Ileana
Department of Rehabilitation Medicine, University of Washington, Seattle, Washington, USA.
Pediatric Rehabilitation Medicine, Seattle Children's Hospital, Seattle, Washington, USA.
Muscle Nerve. 2025 Jan;71(1):29-32. doi: 10.1002/mus.28285. Epub 2024 Oct 28.
The landscape of care for children and adults with neuromuscular disorders (NMDs) is rapidly changing as more disease-modifying treatments (DMTs) become available. These DMTs provide hope and opportunity for evolving phenotypes, though none (yet) are curative. Rehabilitation has been the standard of care for patients with NMDs and should remain so, even with the advent of novel DMTs. An interdisciplinary rehabilitation approach is holistic and comprehensive, addressing functional needs, musculoskeletal complications, pain, durable medical equipment, and bracing needs. This care will continue to be essential for patients who experience impairments and disability, despite receiving DMTs. Additionally, we must consider how to care for a new rapidly expanding cohort of patients aging with NMDs. Children with NMDs are expected to live longer into adulthood; a population that we may not have the workforce to support at this time. At this point, we have the perfect opportunity to reemphasize the importance of rehabilitation in the care of persons with NMDs, while we reexamine historical rehabilitation practices and innovatively deliver services to optimize the effects of these high-cost DMTs.
随着越来越多的疾病修正疗法(DMTs)问世,儿童和成人神经肌肉疾病(NMDs)的护理格局正在迅速变化。这些DMTs为不断演变的表型带来了希望和机遇,尽管目前尚无一种疗法具有治愈性。康复一直是NMDs患者的护理标准,即便新型DMTs出现,也应继续保持这一标准。跨学科康复方法是全面且综合的,涵盖功能需求、肌肉骨骼并发症、疼痛、耐用医疗设备以及支具需求等方面。对于那些尽管接受了DMTs但仍存在功能障碍和残疾的患者而言,这种护理依然至关重要。此外,我们必须思考如何照顾这一迅速扩大的新型NMDs老年患者群体。预计患有NMDs的儿童将存活至成年期;而目前我们可能没有足够的劳动力来支持这一群体。此时此刻,我们有绝佳的机会再次强调康复在NMDs患者护理中的重要性,同时重新审视以往的康复实践,并创新提供服务,以优化这些高成本DMTs的效果。