XXXX : a review of disease pathogenesis and update on future therapies.

@entity1 @entity2640 @entity4240 @entity272
OBJECTIVE: Review the pathogenesis of @entity4240 and provide an update on research currently underway that is aimed at treating and potentially curing this severe @entity272 . DESIGN: Review article. SETTING: Private practice and large teaching hospital. @entity1 : None. MEASUREMENTS: N/A. RESULTS: Currently, @entity1 with @entity4240 are managed with only supportive care. However, there are several promising new treatment avenues that may help @entity1 in the future. These include gene therapy, cell therapy, and protein-based therapy. Each approach offers distinct advantages and disadvantages. CONCLUSIONS: The advances in understanding the molecular basis for @entity2640 over the last few decades has led to significant progress in devising new treatment options. Though many of these approaches remain several years away from regular implementation, it is an exciting time for research in the field.
