BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 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 - Saturday, April 25, 2026
Home » Charcot-Marie-Tooth disease

Articles Tagged with ''Charcot-Marie-Tooth disease''

Neurology/psychiatric

PMP22-RNAi gives long-lasting protection in Charcot-Marie-Tooth

April 10, 2026
No Comments
Researchers from the Cyprus Institute of Neurology and Genetics and collaborators report the development of a gene silencing therapy for Charcot-Marie-Tooth type 1A.
Read More
DNA mutations or genetic disorder concept art
Neurology/psychiatric

Biallelic variants in COX18 identified as cause of Charcot-Marie-Tooth disease

Jan. 16, 2026
No Comments

Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been linked to CMT. These genes encode proteins involved in nerve-specific processes, such as axonal transport, myelination and synaptic transmission, and in general housekeeping pathways. However, the reason underlying why defects in such ubiquitous proteins predominantly affect peripheral nerves remains unclear.


Read More
Drug capsule spilling onto brain

Cycle buying Applied Therapeutics to develop CNS drugs

Dec. 12, 2025
By Lee Landenberger
No Comments
Cycle Group Holdings Ltd. is buying Applied Therapeutics Inc. for a small fraction of the company’s value when it went public in 2019. Cycle is paying $0.088, nearly 9 cents, in cash per share plus a contingent value right (CVR).
Read More
Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

Revir’s RTX-117 designated orphan drug for Charcot-Marie-Tooth disease

Oct. 14, 2025
No Comments
Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.
Read More
Demyelination of a neuron
Neurology/psychiatric

Grant supports evaluation of Neucore’s exosome platform for CMT1A

Sep. 3, 2025
No Comments
Neucore Bio Inc. has received a $350,000 STTR phase I grant from the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health (NIH) to evaluate the company’s targeted exosome platform to deliver an RNA-based therapy to treat Charcot-Marie-Tooth disease type 1a (CMT1A).
Read More
3D dollar sign
Neurology/psychiatric

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 26, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
Read More
3D dollar sign

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 24, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
Read More
Genetic/congenital

CMT2E murine model reveals early axonal damage

March 21, 2025
Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.
Read More
Endocrine/metabolic

Series A financing at Alesta Therapeutics to advance programs in hypophosphatasia and CMT

Jan. 9, 2025
Alesta Therapeutics BV has closed an oversubscribed €65 million (US$67 million) series A financing round designed to support development of its two small-molecule therapeutics.
Read More
Neurology/psychiatric

HDAC inhibitor improves Charcot-Marie-Tooth disease in mice

July 8, 2024
Therapeutics for peripheral neuropathies, such as Charcot-Marie-Tooth (CMT) disease, are still quite limited and focus on managing symptoms. Augustine Therapeutics NV has presented an orally available histone deacetylase 6 (HDAC6) inhibitor, AGT-100216, for the treatment of CMT. AGT-100216 was designed to work through improving axonal regeneration and nerve functional restoration.
Read More
Previous 1 2 3 Next

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for April 24, 2026.
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for April 21, 2026
  • Boy cupping ear with soundwave graphic

    A free gene therapy? Regeneron’s Otarmeni approved for hearing loss

    BioWorld
    Children and adults with a type of congenital hearing loss now have a free treatment option, with the U.S. FDA’s accelerated approval of Regeneron Pharmaceuticals...
  • Illustration of metastatic cancer

    At AACR: Epigenetic fingerprints in metastases track tumor origin

    BioWorld
    When a tumor migrates and colonizes another tissue or organ, it can be identified as a metastasis, but its origin is not always clear. Now, a study based on...
  • Illustration of human face that looks abstract and digital

    AACR 2026: The age of agentic AI in oncology

    BioWorld Science
    New Approach Methodologies (NAMs) for drug development are transforming biomedical research by replacing or complementing animal models. More than 90% of...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • 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 ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing