Cerliponase alfa
- Generic Name
- Cerliponase alfa
- Brand Names
- Brineura
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 151662-36-1
- Unique Ingredient Identifier
- X8R2D92QP1
- Background
Cerliponase alfa is an enzyme replacement treatment for a specific form of Batten disease. It was the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. In...
- Indication
Cerliponase alfa is a treatment for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease to decelerate the progressive motor function decline in patients 3 years of age and older. CLN2 disease is a form of Batten disease, a rare inherited neurodegenerative disorder and is associated with seizures, ataxia, rapid loss of language and motor funct...
- Associated Conditions
- Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
- Associated Therapies
- -
Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
- Conditions
- Interventions
- First Posted Date
- 2021-12-10
- Last Posted Date
- 2023-09-13
- Lead Sponsor
- David L Rogers, MD
- Target Recruit Count
- 5
- Registration Number
- NCT05152914
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
Cerliponase Alfa Observational Study in the US
- Conditions
- Interventions
- First Posted Date
- 2020-07-20
- Last Posted Date
- 2024-01-31
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 35
- Registration Number
- NCT04476862
- Locations
- 🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸Children's Hospital of Orange County, Orange, California, United States
🇺🇸Children's Hospital of Colorado, Aurora, Colorado, United States
Drug Development Updates
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