ReviewActa neurologica Belgica2025
The current status of Charcot-Marie-Tooth disease type 1 A treatment.
Review in Acta neurologica Belgica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Peripheral Myelin Protein-22 and Its Prominence in Charcot-Marie-Tooth Disease.Chemical reviews · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
Charcot-Marie-Tooth disease type 1 A (CMT1A) is the major subtype of hereditary peripheral neuropathies and arises from a 1.5 megabase (Mb) tandem duplication in chromosome 17p11.2-p12 that contains the complete peripheral myelin protein 22 (PMP22) gene. Patients commonly present with progressive weakness and atrophy of the distal muscles, accompanied by hyperalgesia, decreased or absent tendon reflexes, and foot deformities. Current clinical management relies on multidisciplinary supportive care. Recent preclinical studies targeting potential therapeutic strategies for CMT1A have focused on correcting the gene-dose imbalance of PMP22. Notably, PXT3003 has shown phase III clinical efficacy in relieving symptoms and reducing neuropathy, and is expected to be the earliest CMT1A-targeted drug on the market. Gene editing approaches have also shown therapeutic promise in animal models, but off-target effects remain a concern. In addition, the rapid development of induced pluripotent stem cell (iPSC) technology has paved the way for stem cell therapies, which may be a promising therapeutic approach. This article reviews the existing literature on therapeutic strategies for CMT1A and aims to provide a valuable reference for the clinical treatment of CMT1A.
Indexed as
Identifiers
What Socratic holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.