More recently, CLR01 was found to ameliorate significantly the pathologic phenotype in a mouse model of Sanfilippo syndrome type A by a similar relieving of “clogged” lysosomes, allowing them to resume merging with autophagosomes to form autolysosomes and degrade multiple aggregated proteins, including α-synuclein, Aβ, tau, and prion protein26. The gene discussed is MAPT; the disease is mucopolysaccharidosis type 3A.