Indication

Juvenile metachromatic leukodystrophy

Juvenile metachromatic leukodystrophy is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme arylsulfatase A (or rarely saposin B), leading to the toxic accumulation of sulfatides within the central and peripheral nervous systems. Read more about Juvenile metachromatic leukodystrophy

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LENMELDY (atidarsagene autotemcel)

HEMATOPOIETIC AGENTS - HEMATOPOIETIC GENE THERAPY AGENTS·JUVENILE METACHROMATIC LEUKODYSTROPHY·+1

ORCHARD THERAPE

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About Juvenile metachromatic leukodystrophy.

This accumulation causes progressive demyelination and widespread destruction of white matter.

Manifesting typically in children and adolescents between 4 and 16 years of age, initial clinical presentations characteristically include insidious cognitive decline, behavioral changes, declining school performance, and motor difficulties such as ataxia and gait impairment.

As the disease advances, affected individuals experience progressive spasticity, loss of independent ambulation and speech, seizures, and eventual severe neurological decerebration.

rarediseases.org
Juvenile metachromatic leukodystrophy is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme arylsulfatase A (or rarely saposin B), leading to the toxic accumulation of sulfatides within the central and peripheral nervous systems This accumulation causes progressive demyelination and widespread destruction of white matter As the disease advances, affected individuals experience progressive spasticity, loss of independent ambulation and speech, seizures, an...
kennedykrieger.org
Juvenile metachromatic leukodystrophy is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme arylsulfatase A (or rarely saposin B), leading to the toxic accumulation of sulfatides within the central and peripheral nervous systems Manifesting typically in children and adolescents between 4 and 16 years of age, initial clinical presentations characteristically include insidious cognitive decline, behavioral changes, declining school performance, and motor di...
umn.edu
Juvenile metachromatic leukodystrophy is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme arylsulfatase A (or rarely saposin B), leading to the toxic accumulation of sulfatides within the central and peripheral nervous systems
medlineplus.gov
This accumulation causes progressive demyelination and widespread destruction of white matter
rarediseases.org
This accumulation causes progressive demyelination and widespread destruction of white matter
mayoclinic.org
Manifesting typically in children and adolescents between 4 and 16 years of age, initial clinical presentations characteristically include insidious cognitive decline, behavioral changes, declining school performance, and motor difficulties such as ataxia and gait impairment
chp.edu
Manifesting typically in children and adolescents between 4 and 16 years of age, initial clinical presentations characteristically include insidious cognitive decline, behavioral changes, declining school performance, and motor difficulties such as ataxia and gait impairment

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