BRINEURA (cerliponase alfa)
DRUGS TO TREAT NEURONAL CEROID LIPOFUSCINOSIS TYPE 2 (CLN2)·TREATMENT TO SLOW LOSS OF AMBULATION IN LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
BIOMARIN PHARMA
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Treatment to slow loss of ambulation in late infantile neuronal ceroid lipofuscinosis type 2
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, autosomal recessive lysosomal storage neurodegenerative disorder caused by mutations in the *TPP1* gene that lead to deficiency of the enzyme tripeptidyl peptidase 1. Read more about Treatment to slow loss of ambulation in late infantile neuronal ceroid lipofuscinosis type 2
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Common Treatment to slow loss of ambulation in late infantile neuronal ceroid lipofuscinosis type 2 therapies.
BRINEURA (cerliponase alfa)
DRUGS TO TREAT NEURONAL CEROID LIPOFUSCINOSIS TYPE 2 (CLN2)·TREATMENT TO SLOW LOSS OF AMBULATION IN LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS TYPE 2
BIOMARIN PHARMA
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Coming soonLate infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, autosomal recessive lysosomal storage neurodegenerative disorder caused by mutations in the *TPP1* gene that lead to deficiency of the enzyme tripeptidyl peptidase 1.
This enzyme deficiency results in the pathological lysosomal accumulation of autofluorescent ceroid lipopigments within the central nervous system and retina, causing progressive neuronal death and cerebral atrophy.
Typically presenting in children between two and four years of age, the condition manifests as language delay, recurrent seizures, myoclonus, ataxia, visual deterioration, and rapid cognitive and motor regression.
In this population, therapeutic slowing of the loss of ambulation addresses the devastating motor decline that systematically impairs independent gait and eventually leaves affected pediatric patients nonambulatory.
- biomarin.com
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, autosomal recessive lysosomal storage neurodegenerative disorder caused by mutations in the *TPP1* gene that lead to deficiency of the enzyme tripeptidyl peptidase 1 In this population, therapeutic slowing of the loss of ambulation addresses the devastating motor decline that systematically impairs independent gait and eventually leaves affected pediatric patients nonambulatory
- nih.gov
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, autosomal recessive lysosomal storage neurodegenerative disorder caused by mutations in the *TPP1* gene that lead to deficiency of the enzyme tripeptidyl peptidase 1 Typically presenting in children between two and four years of age, the condition manifests as language delay, recurrent seizures, myoclonus, ataxia, visual deterioration, and rapid cognitive and motor regression
- nih.gov
This enzyme deficiency results in the pathological lysosomal accumulation of autofluorescent ceroid lipopigments within the central nervous system and retina, causing progressive neuronal death and cerebral atrophy Typically presenting in children between two and four years of age, the condition manifests as language delay, recurrent seizures, myoclonus, ataxia, visual deterioration, and rapid cognitive and motor regression
- nih.gov
This enzyme deficiency results in the pathological lysosomal accumulation of autofluorescent ceroid lipopigments within the central nervous system and retina, causing progressive neuronal death and cerebral atrophy
- practicalneurology.com
In this population, therapeutic slowing of the loss of ambulation addresses the devastating motor decline that systematically impairs independent gait and eventually leaves affected pediatric patients nonambulatory
- medlineplus.gov
In this population, therapeutic slowing of the loss of ambulation addresses the devastating motor decline that systematically impairs independent gait and eventually leaves affected pediatric patients nonambulatory
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