Monthly Prescribing Reference

Miplyffa Approved for Niemann-Pick Disease Type C

Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...
Nasdaq

Zevra Therapeutics Publishes Long-Term Efficacy and Safety Results for MIPLYFFA in Niemann-Pick Disease Type C

Zevra Therapeutics, Inc. announced the publication of long-term data on the efficacy and safety of MIPLYFFA® (arimoclomol) for treating Niemann-Pick disease type C (NPC) in the journal Molecular ...
MedCity News

FDA Approval Makes a Zevra Drug the First Therapy for Rare and Fatal Metabolic Disease

A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...
Business Wire

Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1

TransportNPC™ is the most comprehensive ongoing controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes for the treatment of Niemann-Pick Disease Type C1 ...
Nasdaq

Zevra Therapeutics Showcases MIPLYFFA® Data at National Niemann Pick Disease Foundation Conference

Zevra Therapeutics presents MIPLYFFA data at the NNPDF Conference, showcasing its FDA-approved treatment for Niemann-Pick Disease type C. Zevra Therapeutics, Inc. announced that its therapy MIPLYFFA® ...
Hosted on MSN

An Overview of Niemann-Pick Disease

Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety of medical ...
The American Journal of Managed Care

5 Rare Diseases That Now Have Their First FDA-Approved Treatments

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease. Since Rare ...
EurekAlert!

Global consortium to study Pick's disease, rare form of early-onset dementia

JACKSONVILLE, Fla. — Pick's disease, a neurodegenerative disease of unknown genetic origin, is a rare type of frontotemporal dementia that affects people under the age of 65. The condition causes ...
Healio

FDA approves Miplyffa for Niemann-Pick disease type C

Please provide your email address to receive an email when new articles are posted on . The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated ...
Monthly Prescribing Reference

Miplyffa Now Available for Niemann-Pick Disease Type C

To start patients on Miplyffa, prescribers can fill out an enrollment form on Miplyffa.com. Miplyffa™ (arimoclomol) is now available for use in combination with miglustat for the treatment of ...
Healio

Miplyffa commercially available for treatment of Niemann-Pick disease type C

Please provide your email address to receive an email when new articles are posted on . Zevra Therapeutics has announced that Miplyffa, the first Nieman-Pick disease type C treatment approved by the ...