demyelinating hereditary motor and sensory neuropathy

ORPHA: 4761161 Treatment Available

Overview

human disease

Available Treatments (1)

DrugFormStatusCountriesLead Time
Eteplirsen
Orphan Cold Chain
IV infusionFDA Approved1

Classification & Codes

Orphanet Code

ORPHA:476116
demyelinating hereditary motor and sensory neuropathy
OrphanetORPHA:476116
Treatments1 drug(s)
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
demyelinating hereditary motor and sensory neuropathy | OrphanDrug