Substance / Medication

Cerliponase alfa

Overview

Clinical overview including description, indications, and evidence base is pending physician authorship or FDA SPL integration. Graph-derived relationship data is available below.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Administration details including route, dosing schedules, preparation instructions, and contraindications are pending physician authorship.

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Chest-sited intraventricular access devices for cerliponase alfa infusion in Batten disease at a single tertiary United Kingdom pediatric center.
Read Jack, Donald Aimee, Rhead Stephanie et al. · J Neurosurg Pediatr · 2025
PMID: 41172362Observational
Intraventricular Cerliponase Alfa Treatment in a Patient with Advanced Neuronal Ceroid Lipofuscinosis Type 2.
Nakashima Saki, Hamada Masashi, Kimura Tomohiko et al. · Intern Med · 2024
PMID: 37926545Case ReportFull text (PMC)
Presymptomatic treatment of classic late-infantile neuronal ceroid lipofuscinosis with cerliponase alfa.
Schaefers J, van der Giessen L J, Klees C et al. · Orphanet J Rare Dis · 2021
PMID: 33990214Case ReportFull text (PMC)
Treatment of Neuronal Ceroid Lipofuscinosis Type 2 with Cerliponase Alfa: A Systematic Review and Single-Arm Meta-Analysis of Two Studies.
Oliveira Juliana Almeida, Saddique Muhammad Nabeel, Qadri Maria et al. · J Child Neurol · 2025
PMID: 41160491Review
Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa.
Schulz Angela, Schwering Christoph, Wibbeler Eva et al. · Front Neurol · 2025
PMID: 40162009OtherFull text (PMC)
Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa.
Guelbert Norberto, Espitia Segura Oscar Mauricio, Amoretti Carolina et al. · Mol Genet Metab Rep · 2024
PMID: 38469103OtherFull text (PMC)
Cerliponase alfa decreases Aβ load and alters autophagy- related pathways in mouse hippocampal neurons exposed to fAβ.
Kose Selma, Cinar Elif, Akyel Hilal et al. · Life Sci · 2024
PMID: 39362589Preclinical

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Risk profile, common and serious adverse effects, contraindications, and drug interactions are pending physician authorship.

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Cerliponase alfa (substance)
SNOMED CT
734039005
UMLS CUI
C4475875

Clinical Data

This intervention maps to 1 entities in the Healos knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Healos clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.