autosomal recessive limb-girdle muscular dystrophy type 2I

MeSH: C564612ORPHA: 34515

Overview

autosomal recessive limb-girdle muscular dystrophy that has material basis in homozygous or compound heterozygous mutation in the gene encoding fukutin-related protein (FKRP) on chromosome 19q13.3

Available Treatments (0)

No treatments linked yet

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

Clinical Presentation

Signs and symptoms associated with autosomal recessive limb-girdle muscular dystrophy type 2I, sourced from HPO and Orphanet clinical annotations.

Elevated circulating creatine kinase concentrationMuscular dystrophyProximal muscle weaknessReduced muscle fiber alpha dystroglycanGeneralized hypotoniaWaddling gaitShoulder girdle muscle weaknessPelvic girdle muscle weaknessAbnormal Achilles tendon morphologyCalf muscle hypertrophyMotor delayDilated cardiomyopathyFrequent fallsScoliosisDifficulty climbing stairsDifficulty runningReduced muscle fiber merosin

Classification & Codes

MeSH Code

C564612

Orphanet Code

ORPHA:34515
autosomal recessive limb-girdle muscular dystrophy type 2I
MeSHC564612
OrphanetORPHA:34515
Treatments0 drug(s)
Symptoms on record17 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO