The question of dual therapy, to gain in efficiency, is arising in SMA. Still, few studies have been published on this subject so far. Early work studying the effects of a double treatment has recently been published:
- an American study in a model mouse with a severe form of SMA shows that, compared to one of the treatments alone, the combination of Spinraza® and Evrysdi® increases the production of SMN, improves their motor function and their lifespan, even when symptoms have already appeared;
- another American study reports the benefits experienced by four patients with SMA and treated with Zolgensma® then Evrysdi® for one month.
Clinical trials, which are not taking place in France, are evaluating the effects of a double treatment on a larger scale:
- the RESPOND study combines Spinraza® with Zolgensma® in 60 infants;
- the MANATEE trial combines Evrysdi® with an anti-myostatin, GYM329 (RO7204239) in 180 children.
