Charcot-Marie-Tooth disease type 4B1

MeSH: C535420ORPHA: 999554 Treatments Available

Overview

Charcot-Marie-Tooth disease type 4 that has material basis in mutation in the gene encoding the myotubularin-related protein-2 (MTMR2)

Available Treatments (4)

DrugFormStatusCountriesLead Time
gabapentin
Oral capsule 100mg, 300mg, 400mg; Oral tablet 600mg, 800mg; Oral solution 250mg/5mLFDA Approved, EMA Approved113d
Baclofen
Oral tablets 10mg, 20mg; Intrathecal injection 0.05mg/mL, 10mg/20mLFDA Approved77d
tizanidine
oral tablet 2mg, 4mg; oral capsule 2mg, 4mg, 6mgFDA Approved45d
pregabalin
oral capsule 25mg, 50mg, 75mg, 100mg, 150mg, 200mg, 225mg, 300mg; oral solution 20mg/mLFDA Approved105d

Classification & Codes

MeSH Code

C535420

Orphanet Code

ORPHA:99955
Charcot-Marie-Tooth disease type 4B1
MeSHC535420
OrphanetORPHA:99955
Treatments4 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO