Spada M, Baron R, Elliott PM, et al.

The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease - A systematic literature review by a European panel of experts.

Mol Genet Metab. 2019;126:212-223.

Ir

Ellaway C.

Paediatric Fabry disease.

Transl Pediatr. 2016;5:37-42. 

Ir

Germain DP, Moiseev S, Suárez-Obando F, et al.

The benefits and challenges of family genetic testing in rare genetic diseases-lessons from Fabry disease.

Mol Genet Genomic Med. 2021;9:e1666. 

Ir