Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study.

doi:10.1016/j.ymgme.2021.02.006

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Article

Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study.

Molecular genetics and metabolism - 1 May 2021

Horovitz Dafne D G, Leão Emília K E A, Ribeiro Erlane M, Martins Ana M, Barth Anneliese L, Neri João I C F, Kerstenetzky Marcelo, Siqueira Ana C M, Ribeiro Bethania F R, Kim Chong A, Santos Francisca C, Franco José F S, Lichtvan Leniza C L, Giuliani Liane R, Rodrigues Maria Do C S, Bonatti Renata C F, Teixeira Thaís B, Gonçalves Alexandra, Lourenço Charles M, Pereira Ane S S, Acosta Angelina X

Abstract excerpt

Patients with mucopolysaccharidosis type VI (MPS VI) present with a wide range of disease severity and clinical manifestations, with significant functional impairment and shortened lifespan. Enzyme replacement therapy (ERT) with galsulfase has been shown to improve clinical and biochemical parameters including patient survival, quality of life and growth. The present study is a resurvey of 34 Brazilian MPS VI...

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PMID: 33678523
DOI: 10.1016/j.ymgme.2021.02.006

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