1. Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships;Al-Sannaa;Orphanet Journal of Rare Diseases,2015
2. Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy;Angelini;Acta Myologica,2012
3. Approved Cellular and Gene Therapy Products (website). https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed May 9, 2023.
4. CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: a systematic review;Babacíc;PLoS One,2019
5. Inhibition of fatty acid amide hydrolase prevents pathology in neurovisceral acid sphingomyelinase deficiency by rescuing defective endocannabinoid signaling;Bartoll;EMBO Molecular Medicine,2020