LENMELDY (atidarsagene autotemcel)
HEMATOPOIETIC AGENTS - HEMATOPOIETIC GENE THERAPY AGENTS·JUVENILE METACHROMATIC LEUKODYSTROPHY·+1
ORCHARD THERAPE
Indication
Infantile metachromatic leukodystrophy
Infantile metachromatic leukodystrophy is a rare, severe, autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, leading to toxic accumulation of sulfatides in the central and peripheral nervous systems. Read more about Infantile metachromatic leukodystrophy
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Coming soonInfantile metachromatic leukodystrophy is a rare, severe, autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, leading to toxic accumulation of sulfatides in the central and peripheral nervous systems.
Manifesting typically in infants and toddlers between 1 and 2 years of age, the condition damages myelin sheaths and causes widespread, progressive white matter degeneration.
Clinically, affected children experience rapid neurodevelopmental regression marked by motor deterioration, gait loss, hypotonia progressing to severe spasticity, peripheral neuropathy, cognitive decline, seizures, and loss of vision and speech.
- medlineplus.gov
Infantile metachromatic leukodystrophy is a rare, severe, autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, leading to toxic accumulation of sulfatides in the central and peripheral nervous systems Manifesting typically in infants and toddlers between 1 and 2 years of age, the condition damages myelin sheaths and causes widespread, progressive white matter degeneration Clinically, affected children experience rapid neurodevelopmental regression m...
- nih.gov
Infantile metachromatic leukodystrophy is a rare, severe, autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, leading to toxic accumulation of sulfatides in the central and peripheral nervous systems
- ulf.org
Infantile metachromatic leukodystrophy is a rare, severe, autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, leading to toxic accumulation of sulfatides in the central and peripheral nervous systems
- medlineplus.gov
Manifesting typically in infants and toddlers between 1 and 2 years of age, the condition damages myelin sheaths and causes widespread, progressive white matter degeneration
- medlineplus.gov
Clinically, affected children experience rapid neurodevelopmental regression marked by motor deterioration, gait loss, hypotonia progressing to severe spasticity, peripheral neuropathy, cognitive decline, seizures, and loss of vision and speech
- nih.gov
Clinically, affected children experience rapid neurodevelopmental regression marked by motor deterioration, gait loss, hypotonia progressing to severe spasticity, peripheral neuropathy, cognitive decline, seizures, and loss of vision and speech
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