What are the treatments available for Lysosomal Disease?
Since there are many different lysosomal disorders, treatments vary considerably. Currently, there is no cure for most lysosomal disorders. Much of the care is focused on managing symptoms of each specific disorder. Treatments may include physical therapy, hearing aids, pain-reducing medication, anti-epileptic drugs and nutritional therapy. Enzyme replacement therapy and bone marrow or stem cell transplantation have proven successful in treating some lysosomal disorders. Research is currently underway to discover new approaches to cure many of the lysosomal diseases, with a particular and hopeful focus on gene therapy. Since these disorders result from an inherited genetic mutation, correcting the genetic defect before the disorder manifests is one anticipated technique that might lead to a cure in the future.
