Ocular cystinosis

Advocacy intelligence hub — real-time data for patient organizations

View full disease page →Search clinical trials →
Download CSV

At a Glance

Live from database

Regulatory Watchboard

4 events
Jun 2025DFT383 in Pediatric Participants With Nephropathic Cystinosis

Novartis Pharmaceuticals — PHASE1

TrialRECRUITING
Apr 2019CYSTEA-BONE Clinical Study

Hospices Civils de Lyon — NA

TrialRECRUITING
Dec 2017

PROCYSBI: FDA approved

PROCYSBI is indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

FDAcompleted
Aug 1994

Cystagon: FDA approved

Treatment of nephropathic cystinosis in adults and children.

FDAcompleted

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

Financial Assistance

2 programs

FINANCIAL LANDSCAPE SUMMARY

2

Total programs

2

Open now

1

Copay cards

Copay Assistance1

Cystagon

Recordati

OpenContact for detailsApply ↗

Patient Assistance Programs1

PROCYSBI

Horizon Therapeutics USA, Inc.

OpenContact for detailsApply ↗

View all support programs on disease page →

Approved Treatments

1 FDA-approved

PROCYSBI

(cysteamine enteric coated)Orphan drug

Horizon Therapeutics USA, Inc.

12.1 Mechanism of Action Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine i...

Approved Dec 2017FDA label ↗

View full treatment details on disease page →

Clinical Trial Landscape

2 active trials
1Phase 1
1N/A
2Total recruiting
Search clinical trials for Ocular cystinosis

Recent News & Research

No recent news articles indexed yet for Ocular cystinosis.
Search PubMed for Ocular cystinosis

Browse all Ocular cystinosis news →

Specialist Network

Top 6 by expertise

View all Ocular cystinosis specialists →

Quick Actions