Because of the intricate architecture of brain tissue and the blood–brain barrier's selective permeability, the brain continues to be one of the most difficult therapeutic targets. Nanomedicines are being extensively researched as means of delivering therapeutics into the brain, despite their relatively large size when compared to small molecules and nucleic acids. Here, we expand on the potential applications of nanomedicines for treating uncommon neurodevelopmental disorders, utilizing the case of Globoid cell leukodystrophy (Krabbe disease) as a framework. The lessons gained from studying nanoparticle delivery to the brain in the context of Globoid cell leukodystrophy (GCL) can have a wider impact on the treatment of various other neurodevelopmental and neurodegenerative disorders because Globoid cell leukodystrophy is a monogenetic disorder and lysosomal storage disease affecting the nervous system. In this review, we provide an overview of the genetic foundation and epidemiology of Globoid cell leukodystrophy,talk (GCL) about the disease's current in vivo and in vitro models, and address approved and clinically developed therapeutic approaches. Next, we go into more detail about the difficulties in getting particles to the brain, focusing on ways to get nanomedicines past the blood-brain barrier. We emphasize the use of nanoparticles in delivering.