Lysosomal storage disorders
Lysosomal storage disorders are individually rare but collectively common, affecting 1 in around 7,000 people. The more than 50 disorders identified so far share little by way of symptoms, but the one thing that unites them is that they all involve some fault with the lysosome — the cell’s recycling centre. The study of these diseases is not only leading to better treatments, but also revealing many of the secrets of this underappreciated organelle.
For more on lysosomal storage disorders from nature.com, see: nature.com/subjects/lysosomes
Nature is pleased to acknowledge financial support from Alexion Pharmaceuticals, Inc. and Ultragenyx Pharmaceutical Inc., and grants from Shire plc and BioMarin Pharmaceuticals, Inc. in producing this Outlook. As always, Nature retains sole responsibility for all editorial content.
