An Open-Label, Multicenter, Ascending Dose Study Of The Tolerability And Safety Of Recombinant Human Acid Sphingomyelinase (rhASM) In Patients With Acid Sphingomyelinase Deficiency (ASMD)
ID Number 12-1897Principal Investigator(s)
Melissa P. Wasserstein
Department(s) or Division(s)
Genetics and Genomic Sciences
This is a study designed to find out if a drug called recombinant human acid sphingomyelinase (rhASM) is safe to give to humans. This drug has been developed to treat a disease called Niemann Pick Disease type B or Acid Sphingomyelinase Deficiency (ASMD). If the safety of the drug is shown additional studies will be carried out to see of it reverses and prevents the symptoms of ASMD.
Natalie Lippa, MS, CGC
Recruiting Patients: No