California offers lifeline to 17 troubled hospitals

Dyne Ther­a­peu­tics shared promis­ing clin­i­cal tri­al up­dates from two of its ex­per­i­men­tal RNA med­i­cines for my­oton­ic dy­s­tro­phy type 1 (DM1) and Duchenne mus­cu­lar dy­s­tro­phy. The

Almost half of adults and children with neurofibromatosis type 1 (NF1) responded to the investigational MEK inhibitor mirdametinib, a large multicenter study of the rare