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gangliosidosis GM3

ORPHA: 79257

Contents

  1. 1. Overview
  2. 2. Available Treatments
  3. 3. Classification & Codes

Overview

gangliosidosis that is characterized by neurodegeneration and mild skeletal changes and has material basis in mutation in the gene encoding beta-galactosidase-1

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Classification & Codes

Orphanet Code

ORPHA:79257
gangliosidosis GM3
OrphanetORPHA:79257
Treatments0 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO