Quality of life for patients with XLMTM and their caregivers

Living with XLMTM can pose a substantial burden on quality of life

  • Patients with X-linked myotubular myopathy (XLMTM) who survive the first year of life face an increasing need for ventilation and optimization of respiratory care (eg, tracheostomy) as they age and experience continued mobility limitations.1,2 These pose considerable challenges, including speech and communication difficulties, limitations on social activities, and anxiety over cost of care and ventilator failure.2,3
  • Caregivers may face difficult decisions around care options, the inability to work due to 24-hour care needs, isolation, and other financial and emotional burdens related to caring for someone with a life-threatening disease.2

Early recognition of symptoms and diagnosis of XLMTM is important for effective disease management.


1. Dowling JJ, et al. In: Adam MP, et al, eds. GeneReviews®. 2002. 2. Beggs AH, et al. Muscle Nerve. 2018;57(4):550-560. 3. Herman GE, et al. J Pediatr. 1999;134(2):206-214.


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