5 Lysosomal Storage Diseases with Orphan Drugs in Development

In Clinical Development by Tim Peplow

Lysosomal storage diseases are currently a major area of focus for orphan drug development. This group of over 50 rare inherited disorders, each of which are caused by deficiencies of specific enzymes required in the breakdown of carbohydrates, has attracted interest because although enzyme replacement therapy (ERT) remains a valid treatment option in some cases, the opportunity for improvement is still enormous. Here are 5 lysosomal storage diseases with promising orphan drugs in development. Click Next to read them all.


Download the brochure for the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.