Spinal muscular atrophy with respiratory distress type 1

Orpha code: 98920OMIM code: 604320

Definition

Spinal muscular atrophy with respiratory distress type 1 is a rare genetic motor neuron disease characterized by severe respiratory distress/respiratory failure in association with diaphragmatic eventration and palsy, as well as progressive, symmetrical, distal-to-proximal muscle weakness and atrophy (in lower limbs especially). Patients typically have a history of intrauterine growth retardation, low birth weight, feeble cry, weak suck and failure to thrive and present with inspiratory stridor, recurrent episodes of dyspnea or apnea, cyanosis and absent deep tendon reflexes. Kyphosis/scoliosis, foot deformities and joint contractures are frequently associated features.

Disease data
Classification

Disease

Synonyms
Autosomal recessive distal spinal muscular atrophy type 1
Autosomal recessive distal spinal muscular atrophy type 1
Autosomalny recesywny dystalny rdzeniowy zanik mięśni typu 1
Autosomalny recesywny rdzeniowy zanik mięśni z niewydolnością oddechową
dHMN6
dSMA1
Dystalna dziedziczna neuropatia ruchowa typu 6
Dystalna-HMN typu 6
Rdzeniowy zanik mięśni przepony
SIANRF
SMARD1
Autosomal recessive spinal muscular atrophy with respiratory distress
Diaphragmatic spinal muscular atrophy
Distal hereditary motor neuropathy type 6
Distal-HMN type 6
SIANRF
SMARD1
Severe infantile axonal neuropathy with respiratory failure type 1
dHMN6
dSMA1
ORPHA code
98920
OMIM code
604320
ICD10 code
G12.2
ICD11 code
-

No additional description.

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