Acid Sphingomyelinase Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Acid Sphingomyelinase Deficiency trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and on…
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (e…
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingom…
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and on…