BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Monday, December 8, 2025
See today's BioWorld ScienceSee today's BioWorld
Home » Two enzymatic modulators could alleviate Gaucher disease
To read the full article sign up for free or sign in.
Endocrine/metabolic

Two enzymatic modulators could alleviate Gaucher disease

Aug. 26, 2024
By Mar de Miguel
People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.
BioWorld BioWorld Science Science Endocrine/metabolic Genetic/congenital Neurology/psychiatric

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for Dec. 5, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for Dec. 5, 2025.
  • Mesothelin is biomarker, potential target in arthritic bone damage

    BioWorld Science
    In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences...
  • 3D rendering of prion structure

    Epigenetic technology could eliminate misfolded prion proteins

    BioWorld Science
    The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal...
  • Novo Nordisk semaglutide pill

    CTAD 2025: Diagnosing semaglutide’s failure in Alzheimer’s trials

    BioWorld
    A little over a week after announcing that the Evoke and Evoke+ studies failed to show that oral semaglutide could slow cognition decline in patients with...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • NME Digest
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing