Niemann-Pick Disease Type C
May 28, 2025
Everything You Need to Know About Niemann-Pick Disease – From Types to Therapies
Niemann-Pick Disease (NPD) is a cluster of rare, genetically inherited lysosomal storage disorders characterized by the pathological accumulation of lipids within various organs. This buildup leads to progressive multisystem dysfunction, with severity and progression varying significantly across subtypes. While ind...
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May 21, 2025
Unveiling Lysosomal Storage Disorders: Exploring Rare Diseases Impacting Millions Worldwide
Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...
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May 02, 2025
New Era in Niemann-Pick Disease: A Comparative Review of MIPLYFFA and AQNEURSA
Niemann-Pick disease type C (NPC) is a rare, aggressive, and life-threatening neurovisceral disorder with a profound impact on both patients and the healthcare system. Caused by mutations in the NPC1 or NPC2 genes, NPC disrupts intracellular lipid transport, leading to toxic accumulation of cholesterol and other li...
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Oct 01, 2024
IntraBio’s AQNEURSA Niemann-Pick Disease Approval; FDA Approves Novel Schizophrenia Drug After 35 Years; Selpercatinib Gets FDA Nod for RET-Mutated MTC; DUPIXENT Receives First-Ever COPD Approval; Pfizer Withdraws OXBRYTA for Sickle Cell Disease from Global Market
IntraBio's AQNEURSA Receives Historic FDA Approval for Niemann-Pick Disease Type C Treatment IntraBio Inc. has received approval from the FDA for AQNEURSA (levacetylleucine), marking a significant milestone in the treatment of neurological manifestations of Niemann-Pick disease type C in both adults and pe...
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Sep 24, 2024
Zevra’s MIPLYFFA Niemann-Pick Disease Approval; FASENRA Approved for Eosinophilic Granulomatosis; Sanofi’s SARCLISA Gets Multiple Myeloma Approval; RYBREVANT Combo Gets FDA Nod for EGFR Lung Cancer; UCB’s BIMZELX Receives Multiple Approval from the FDA
FDA Greenlights Zevra Therapeutics’ MIPLYFFA for Niemann-Pick Disease Type C Zevra Therapeutics, Inc. has announced that the FDA has approved MIPLYFFA (arimoclomol) capsules as the first oral treatment for Niemann-Pick disease type C. Indicated for use alongside miglustat, MIPLYFFA is intended for both adult and...
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