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BioWorld - Thursday, July 30, 2026
Home » Keywords » Charcot-Marie-Tooth disease

Items Tagged with 'Charcot-Marie-Tooth disease'

ARTICLES

Neurology/psychiatric

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

April 10, 2026
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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.
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DNA mutations or genetic disorder concept art
Neurology/psychiatric

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

Jan. 16, 2026
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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.


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Drug capsule spilling onto brain

Cycle buying Applied Therapeutics to develop CNS drugs

Dec. 12, 2025
By Lee Landenberger
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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).
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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
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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.
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Demyelination of a neuron
Neurology/psychiatric

Grant supports evaluation of Neucore’s exosome platform for CMT1A

Sep. 3, 2025
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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).
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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.
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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.
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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.
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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.
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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.
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More Articles Tagged with 'Charcot-Marie-Tooth disease'

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