Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases
The complexity and organization of the central nervous system (CNS) is widely modulated by the presence of the blood–brain barrier
Tag: Niemann Pick Disease
Cyclo Therapeutics Inc. Commences Commercial-Scale Manufacturing for Trappsol® Cyclo™
On July 27, the Cyclo Therapeutics, a clinical stage biotechnology firm dedicated to developing life-changing medicines through science and innovation
Recent reviews on cyclodextrins against SARS-CoV-2
Recent reviews emphasized the role of membrane lipids in the infectivity mechanism of SARS-COV-2. Cholesterol-rich parts of cell membranes serve
Mallinckrodt gave up further development of adrabetadex (VTS-270; HPBCD)
Mallinckrodt Pharmaceuticals has reached an agreement to divest and transfer the Investigational New Drug (IND) application for experimental drug adrabetadex
Weakly acidic carboxy group-grafted β-cyclodextrin-threaded acid-degradable polyrotaxanes for modulating protein interaction and cellular internalization
To improve the therapeutic potential of β-cyclodextrin (β-CD)-threaded acid-degradable polyrotaxanes (β-CD PRXs) in cholesterol-related metabolic disorders, the effect of carboxylation
Cyclo Therapeutics Announces Design of Pivotal Phase 3 Study Evaluating Trappsol® Cyclo™ in Niemann-Pick Type C1
Cyclo Therapeutics, Inc., a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and
Central nervous system delivery of molecules across the blood-brain barrier
Therapies targeting neurological conditions such as Alzheimer’s or Parkinson’s diseases are hampered by the presence of the blood-brain barrier (BBB).
Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model Mice
Niemann–Pick disease type C (NPC) is a recessive hereditary disease caused by mutation of the NPC1 or NPC2 gene. It is characterized by abnormality
Why HPGCD is better for NPC treatment compared to HPBCD?
Niemann-Pick disease type C (NPC) is a lysosomal storage disorder with disrupted intracellular cholesterol trafficking. HP-β-CD is a cholesterol solubilizer
Cyclo Therapeutics Meets Primary Efficacy Endpoint in Phase 1/2 Trial from Intravenous Trappsol® Cyclo™ in Rare Disease Niemann-Pick Type C1 (NPC1)
100% of patients who completed the trial improved or remained stable, and 89% met the efficacy outcome measure of improvement