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Autor:
Amal AlNaimi, Sara G. Hamad, Reem B. A. Mohamed, Tawfeg Ben‐Omran, Khalid Ibrahim, Mahmoud F. El‐Said Osman, Mutasim Abu‐Hasan
Publikováno v:
Pediatric Pulmonology. 58:1004-1011
Spinal muscular atrophy (SMA) is an inherited progressive neuromuscular disorder characterized by generalized hypotonia, respiratory failure and early death. The introduction of gene replacement therapy (GRT) modified the natural history of the disea