Researchers have studied the levels of endogenous circulating myostatin in patients with SMA, at a time when several anti-myostatin drugs are being tested:
- myostatin and follistatin were measured before and after treatment with an innovative compound,
- 25 patients, including 13 with type 1 SMA, took part in this retrospective study,
- before treatment, myostatin levels correlated with age, weight, type of SMA and motor function,
- although motor performance scores improved objectively under nusinersen, little or no variation in myostatin was noted.
However, further large-scale studies will be needed to gain a better understanding of the impact of innovative treatments on myostatin levels in SMA and to refine the inclusion criteria for patients in trials combining anti-myostatins.
