The promise of gene therapy is clear, even if its route to effective medicine has been anything but. LPLD was a good candidate for gene therapy because it is a single-gene disorder. Gene therapy offers the promise of curing inherited disease, but the most notable successes have been with respect to single-gene disorders, which usually correspond with rare diseases.
Glybera is currently seeking US approval. If you are one of the one-in-a-million that suffer from LPLD, you probably have been following Glybera, but if you think it could help you, talk to your doctor.