Acid Sphingomyelinase Deficiency (Niemann-Pick Disease types A and B)
For information on Xenpozyme, approved for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, visit XENPOZYME® (olipudase alfa-rpcp).
Niemann-Pick Disease type C
AQNEURSA™ (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
AQNEURSA is the only FDA-approved stand-alone therapy for the treatment of NPC and is available now.
For more information visit aqneursa.com
AQNEURSA Cares Program Information
MIPLYFFA™ is the first FDA-approved treatment for Niemann-Pick disease type C (NPC). MIPLYFFA™ is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
For information on MIPLYFFA™ visit miplyffa.com
Each company offers a patient support program that can help with insurance questions, prior authorizations, copay assistance, and coordinating shipments from the specialty pharmacy. Your prescribing physician typically starts the enrollment process. If you run into barriers, NNPDF can help you understand next steps.
Treatments not yet approved may be available through an Expanded Access Program.
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