Yargesa™ (miglustat)
| Full Name | Yargesa™ (miglustat) |
| Drug | Yargesa |
| Manufacturer | Edenbridge Pharmaceuticals |
| 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 | Yargesa Enrollment Form |
| Phone Number | 800-240-9572 |
| Fax Number | 877-220-7581 |
| Patient Resources | National Niemann Pick Disease Foundation |