infantile-onset X-linked spinal muscular atrophy

MeSH: C535380ORPHA: 1145

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 infantile-onset X-linked spinal muscular atrophy, sourced from HPO and Orphanet clinical annotations.

AreflexiaPoor suckDegeneration of anterior horn cellsSkeletal muscle atrophyEMG: neuropathic changesAbnormal muscle fiber morphologyAbnormal anterior horn cell morphologyHigh palateShort ribsCupped ribsInterphalangeal joint contracture of fingerMyopathic faciesRespiratory insufficiencyRespiratory distressAnarthriaArthrogryposis multiplex congenitaElbow flexion contractureHip contractureProximal muscle weaknessKnee flexion contractureAnkle flexion contractureSpinal muscular atrophyFloppy infantInflammatory myopathyBone fractureCryptorchidismGeneralized hypotoniaTongue fasciculationsPotter faciesDistal muscle weaknessAbnormal periventricular white matter morphologyKyphoscoliosisGeneralized muscle weaknessMotor polyneuropathyMildly elevated creatine kinaseWeakness of facial musculatureCognitive impairment

Classification & Codes

MeSH Code

C535380

Orphanet Code

ORPHA:1145
infantile-onset X-linked spinal muscular atrophy
MeSHC535380
OrphanetORPHA:1145
Treatments0 drug(s)
Symptoms on record37 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO