@article{velho_sperb-ludwig_schwartz_2015,  title={New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals},  volume={87},  ISSN={0001-3765},  url={https://doi.org/10.1590/0001-3765201520140711},  DOI={10.1590/0001-3765201520140711},  number={2},  journal={Anais da Academia Brasileira de Ciências},  publisher={Academia Brasileira de Ciências},  author={VELHO, RENATA V. and SPERB-LUDWIG, FERNANDA and SCHWARTZ, IDA V.D.},  year={2015},  month={Aug},  pages={1375–1388} }