Efficacy of the Janus kinase 1/2 inhibitor ruxolitinib in the treatment of vasculopathy associated with TMEM173-activating mutations in 3 children.

Frémond M,Rodero M,Jeremiah N,Belot A,Jeziorski E,Duffy D,Bessis D,Cros G,Rice G,Charbit B,Hulin A,Khoudour N,Caballero C,Bodemer C,Fabre M,Berteloot L,Le Bourgeois M,Reix P,Walzer T,Moshous D,Blanche S,Fischer A,Bader-Meunier B,Rieux-Laucat F,Crow Y,Neven B

Source :

2016 Aug 21