Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.

2025
https://researcherprofiles.org/profile/679811745
40663813
Mengel E, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg SW, Harmatz P, Hennermann JB, Héron B, Maier EM, Santra S, Sharma R, Tylki-Szymanska A, Cording M, Himmelstrup L, Guenther S, Dali CÍ