We report a patient with mucopolysaccharidosis type VI, on long-term (2 years, 6 months) galsulfase enzyme replacement home therapy. Results support the efficacy and safety benefits, with additional advantage of home therapy to minimize the risk of community-transmitted infections at this time of the COVID-19 pandemic. Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal storage disorder caused by a deficiency of the N-acetylgalactosamine 4 sulfatase enzyme (also known as arylsulfatase B or ASB), which results in the accumulation of the glycosaminoglycan (GAG) dermatan sulfate (DS) in different tissues of the body. 1 The disease is associated with high morbidity and reduced life expectancy, 2,3 although its incidence is lower than that of other MPS (1/455 000 live births). 4 In Monte Santo, Brazil, the prevalence is estimated at 20/1 000 000, probably as a result of a founding effect and inbreeding, with a single homozygous mutation present (p.H178L). 5 No studies reporting the prevalence of this disease are available in Peru or other Latin American countries. The N-acetylgalactosamine 4 sulfatase enzyme is found predominantly not only in the skin, but also in tendons, blood vessels, airways, and heart valves. 6 Studies have shown that DS accumulation creates an inflammatory response through the tumor necrosis factor (TNF) pathway, resulting in chondrocyte apoptosis and ensuing progressive arthropathy. 7,8 It affects bone, cartilage, liver, spleen, ligaments, joints, heart valves, airways, meninges, and corneas. As a result of the accumulation of DS in the different tissues, clinical signs and symptoms are multisystemic and heterogenous. There is evidence of growth stunting, coarse facies, thick hair, skeletal deformities, frequent upper airway infections, hepatosplenomegaly, hearing loss, sleep apnea, and stiff joints. 2 Other anatomical abnormalities and heart valve dysfunction have been reported in all patients. 3 Patients with MPS VI generally appear healthy at birth, with symptoms usually manifesting in early infancy as a result of increased GAG concentration in the cells. The clinical

A patient with mucopolysaccharidosis type VI, on enzyme replacement therapy (galsulfase) for more than 6-years with good adherence. Urine glycosaminoglycans became stable, cardiopulmonary progression is slow, motor skills have improved, pain is controlled and no adverse reactions were reported. These support the benefits of galsulfase, as it slows disease progression. Key Clinical MessageA patient on enzyme replacement therapy (galsulfase) for more than 6-years. Supporting the long-term benefits of galsulfase as an option to modify the natural history and the quality of life in patients with Mucopolysaccharidosis VI.