Follow the Follistatin

Attempts to treat muscular dystrophy have generally followed one of two paths, neither of which has yet reached the market. One option is delivering the dystrophin protein that is missing or dysfunctional via gene or cell therapy. The other is inhibiting myostatin, a negative regulator of muscle growth.¹ Now a study published in the Proceedings of the National Academy of Sciences combines the two approaches, using gene therapy to deliver follistatin, a myostatin inhibitor, to diseased muscle.

There are no approved drugs for the disease, and last month's discontinuation of Wyeth's MYO-029 antimyostatin antibody was just the latest stumble for companies in search of a treatment for muscular dystrophy (MD)...