Adults with lysosomal storage diseases in the undiagnosed diseases network.
Changrui XiaoMary KoziuraHeidi CopeRebecca SpillmanKhoon TanFuki M HisamaCynthia J TifftCamilo ToroPublished in: Molecular genetics & genomic medicine (2022)
LSDs figure highly in the differential diagnosis of neurometabolic disorders in pediatric onset progressive diseases. In adults, their subtle initial presentations overlap with symptoms of more common disorders and less practitioner awareness may lead to prolonged diagnostic challenges.