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Brand Name

Miplyffa

Generic Name

Arimoclomol

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FDA approval date: September 20, 2024

Form: Capsule

What is Miplyffa (Arimoclomol)?

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

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Related Latest Advances

Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?

Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?

Journal: Expert review of neurotherapeutics

Published: April 20, 2026

Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US early access program over a 4-year period.

Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US early access program over a 4-year period.

Journal: Molecular genetics and metabolism

Published: April 13, 2026

Effects of Lysine Deacetylation Inhibition Alone or in Combination With Arimoclomol on TDP-43 Proteinopathy.

Effects of Lysine Deacetylation Inhibition Alone or in Combination With Arimoclomol on TDP-43 Proteinopathy.

Journal: Journal of neurochemistry

Published: December 17, 2025

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