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Authors
Wasserstein, Melissa P. http://orcid.org/0000-0003-2031-7817
Lachmann, Robin
Hollak, Carla
Barbato, Antonio
Gallagher, Renata C.
Giugliani, Roberto
Guelbert, Norberto Bernardo
Hennermann, Julia B.
Ikezoe, Takayuki
Lidove, Olivier
Mabe, Paulina
Mengel, Eugen
Scarpa, Maurizio
Senates, Ebubekir
Tchan, Michel
Villarrubia, Jesus
Thurberg, Beth L.
Yarramaneni, Abhimanyu
Armstrong, Nicole M.
Kim, Yong
Kumar, Monica
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Clinical Trials BETA
Clinical trials referenced in this document:
nct02004691 at ClinicalTrials.govDocuments that mention this clinical trial
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
https://doi.org/10.1186/s13023-023-02983-0
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Funding
Funding for this research was provided by:
Sanofi
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Article History
Received: 7 June 2023
Accepted: 18 November 2023
First Online: 2 December 2023
Declarations
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: Local 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.
: Not applicable.
: 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.
