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Miglustat

Full Name miglustat
Drug Miglustat
Manufacturer Generic
Route of Administration Oral
Site of Care Home or Healthcare Facility
Approved Indication Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option

Use in combination with MIPLYFFA™ (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older

Disease Gaucher Type I Disease, Niemann-Pick disease type C (NPC)
Therapeutic Area Neurology, Genetics
Enrollment Form Link Miglustat Enrollment Form
Phone Number 800-240-9572
Fax Number 877-220-7581