Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial.
Melissa P WassersteinRobin LachmannCarla HollakAntonio BarbatoRenata C GallagherRoberto GiuglianiNorberto Bernardo GuelbertJulia B HennermannTakayuki IkezoeOlivier LidovePaulina MabeEugen MengelMaurizio ScarpaEbubekir SenatesMichel TchanJesus VillarrubiaBeth L ThurbergAbhimanyu YarramaneniNicole M ArmstrongYong KimMonica KumarPublished in: Orphanet journal of rare diseases (2023)
Treatment with olipudase alfa is well tolerated and reduces manifestations of chronic ASMD with sustained efficacy. Trial registration NCT02004691 registered 9 December 2013, https://clinicaltrials.gov/ct2/show/NCT02004691.