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.

"Declarations Ethics approval and consent to participateLocal Institutional Review Boards (IRB) at both study sites approved the protocol; corresponding author’s IRB: Biomedical Research Alliance of New York (BRANY, Lake Success, NY) and patients/parents provided written informed consent prior to patient screening. All clinical data were de-identified. The study adhered to the principles set out in the Declaration of Helsinki. Consent for publicationNot applicable. Competing interestsMW: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa and has received travel reimbursement and consulting fees from Sanofi. AB: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa, received honoraria for lectures, advisory boards, meetings, and travel support from Sanofi and Takeda Shire. RCG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. RG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received honoraria, consulting fees, speaker fees, and travel reimbursement from Sanofi. NBG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. JBH: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received speaker fees from Sanofi. CH: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. TI: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. RL: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received consulting fees and travel reimbursement from Sanofi. OL: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. PM: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa and has received consulting fees, speaker fees, and travel reimbursement from Sanofi. EM: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received consulting fees and honoraria from Sanofi. MS: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. ES: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. MT: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. JV: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. BLT, AY, NMA, YK, and MK were/are employees of Sanofi (or were at the time of the study) and own stock in the company. Competing interests MW: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa and has received travel reimbursement and consulting fees from Sanofi. AB: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa, received honoraria for lectures, advisory boards, meetings, and travel support from Sanofi and Takeda Shire. RCG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. RG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received honoraria, consulting fees, speaker fees, and travel reimbursement from Sanofi. NBG: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. JBH: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received speaker fees from Sanofi. CH: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. TI: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. RL: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received consulting fees and travel reimbursement from Sanofi. OL: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. PM: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa and has received consulting fees, speaker fees, and travel reimbursement from Sanofi. EM: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa; has received consulting fees and honoraria from Sanofi. MS: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. ES: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. MT: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. JV: Principal Investigator in the Sanofi sponsored ASCEND trial of olipudase alfa. BLT, AY, NMA, YK, and MK were/are employees of Sanofi (or were at the time of the study) and own stock in the company."

"Funding Sanofi was the study sponsor and was involved in the study design, data collection, data analysis, interpretation of data, and collaboration with authors on the writing of the manuscript. All authors had full access to the trial data and were responsible for the data accuracy and interpretation of the results. The corresponding author had final responsibility for the submission of the manuscript."

"Trial registration NCT02004691 registered 9 December 2013, https://clinicaltrials.gov/ct2/show/NCT02004691"