ABSTRACT

Lysosomes are spherical membrane-bound vesicles containing more than 60 different enzymes within the organelle’s membrane and its lumen. Lysosomal storage diseases (LSDs) include among others sphingolipidoses with the accumulated products being, e.g., gangliosides, mucopolysaccharidoses, glycoprotein lysosomal storage disorders, and glycogen storage disease type II also known as Pompe disease. Treatment of LSDs may also benefit from a combination of the different therapy forms making use of synergetic effects. Galactosialidosis that belongs to the LSDs, and that originally was classified as a variant of GM1-gangliosidosis has meanwhile turned out to be a disease distinctive from the latter. To sum up it can be expected that in the future more effective therapies may be available for treating LSDs.