- Positive interim results in infants and young children with ENPP1 Deficiency showed improvements from baseline in multiple measures of disease, including survival, heart function, and stabilization ...
Marks a step forward in broadening treatment options for patients with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and generalized myasthenia gravis (gMG) in ...