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Tag: Niemann-Pick disease type C

September 7, 2026 CD as API / Pharma applications

Reducing Cholesterol with β-CDP: Insights for NPC and AD

Niemann–Pick disease type C (NPC) and Alzheimer’s disease (AD) are neurodegenerative disorders characterised by cognitive dysfunction and neurological impairment. In

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March 2, 2026 CD as API / Pharma applications

Beren Therapeutics’Adrabetadex Received FDA Acceptance for infantile-onset Niemann Pick Disease

Beren Therapeutics Announced FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C. Beren Therapeutics

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October 13, 2025 CD as API / Pharma applications

JIC Venture Growth Investments Invests in Beren Therapeutics P.B.C. to Advance Innovative, Cyclodextrin-based Therapeutics

JIC Venture Growth Investments (VGI) announced that it has invested in Beren Therapeutics P.B.C. (“Beren Therapeutics”). Beren Therapeutics is developing

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September 22, 2025 CD as API / Pharma applications

7 of 9 patients showed stabilization or improvement: preliminary data from ongoing Phase 3 TransportNPC™ clinical trial

Rafael Holdings, Inc. announced that data from Cyclo Therapeutics’ Phase 3 TransportNPC™ open-label, single-arm sub-study evaluating Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) for Niemann-Pick

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August 27, 2024 CD as API / Pharma applications

Rafael Holdings and Cyclo Therapeutics merge to advance Niemann-Pick drug as FDA rival decision nears

Rafael Holdings and Cyclo Therapeutics on Thursday announced a merger agreement as they look to spur development of their Niemann-Pick

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August 8, 2024 CD as API / Pharma applications

Cyclodextrins as therapeutic drugs for treating lipid metabolism disorders

This study sought to systematically compare the efficacy and mechanism of cyclodextrins as drug interventions in lipid metabolism diseases, potentially

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April 8, 2024 CD as API / Pharma applications

Alterations in Proteostasis Mechanisms in Niemann–Pick Type C Disease

Niemann–Pick Type C (NPC) represents an autosomal recessive disorder with an incidence rate of 1 in 150,000 live births, classified

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January 9, 2024 CD as API / Pharma applications

Merger of Cyclo Therapeutics and Applied Molecular Transport

Cyclo Therapeutics, (Cyclo) a clinical stage company focused on developing therapies for rare and neurological diseases with limited treatment options,

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December 5, 2023 CD as API / Pharma applications

Molecular Mind Games: The Medicinal Action of Cyclodextrins in Neurodegenerative Diseases

This work presents the most recent advances in the research and clinical use of HPβCD against Niemann–Pick disease, a congenital

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October 4, 2023 CD as API / Pharma applications

The background story on how the HPBCD became and orphan drug (Hommage’ a Families, Parents)

The Pulitzer Prize-winning Wall Street Journal science- and health writer, Amy Dockser Marcus, in her new book „We the Scientists”

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