Enzyme replacement therapy:
requires the injection of manufactured enzyme into a patient in whom that specific enzyme is missing. This form of therapy has been most successful in patients affected by Gaucher disease. Clinical trials of enzyme replacement therapy are being planned in the near future for other lysosomal storage disorders.

and

Gene replacement therapy:
aims at introducing the appropriate gene into a patient to enable the correct enzyme to be produced in the patient's own cells.

Both of these therapies will enable the stalled recycling process to start and work normally, thereby reversing the storage-causing pathology. The effectiveness of all these therapies would benefit from presymptomatic diagnosis, preferably at birth, with the appropriate therapy started soon after to maximise benefits.

The success of enzyme replacement therapy for Gaucher disease has offered an improved quality of life for patients, their families and carers, and has given HOPE OF A SOLUTION to patients affected by other lysosomal storage disorders.

Therapy also offers significant cost-benefit advantages. It is estimated that the annual medical costs involved in currently managing each lysosomal storage disorder patient can run into hundreds of thousands of dollars per annum. Over several years, therapy could represent a cost saving of millions of health dollars.