CLN2 disease

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At a Glance

Live from database

Regulatory Watchboard

2 events
May 2023A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

Tern Therapeutics, LLC — PHASE1, PHASE2

TrialRECRUITING
Nov 2021Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

David L Rogers, MD — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

Patient Assistance Programs1

Brineura

BioMarin Pharmaceutical Inc.

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Approved Treatments

1 FDA-approved

Brineura

(CERLIPONASE ALFA)Orphan drugstandard

BioMarin Pharmaceutical Inc.

FDA label ↗

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Clinical Trial Landscape

2 active trials
2PHASE1, PHASE2
2Total recruiting
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Recent News & Research

No recent news articles indexed yet for CLN2 disease.
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Specialist Network

Top 6 by expertise

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