Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial.

Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, Parini R, Hughes D, Pastores GM, Lau HA, Al-Sayed MD, Raiman J, STRIVE Investigators , Yang K, Mealiffe M, Haller C