Fabry disease

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At a Glance

Live from database

3FDA treatments

46Recruiting trials

⚡ Live Alerts

PAN Foundation — Fabry disease status changedApply now ↗

Regulatory Watchboard

10 events

Apr 2026Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

Chiesi Italia

TrialNOT YET RECRUITING

Apr 2026Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

University Hospital, Toulouse

TrialNOT YET RECRUITING

Feb 2026A Study of Patients With Fabry Disease (US Specific)

Amicus Therapeutics

TrialRECRUITING

Jan 2026A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Amicus Therapeutics — PHASE3

TrialRECRUITING

Jan 2026Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease

Núcleo de Apoio à Investigação Clínica - FMUP

TrialNOT YET RECRUITING

Nov 2025Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

Yonsei University

TrialRECRUITING

Nov 2025A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

Takeda — NA

TrialRECRUITING

Oct 2025Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT

General University Hospital, Prague — NA

TrialENROLLING BY INVITATION

Jul 2025A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

Chiesi Farmaceutici S.p.A. — PHASE2, PHASE3

TrialRECRUITING

Jun 2025Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

IRCCS Azienda Ospedaliero-Universitaria di Bologna — NA

TrialRECRUITING

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

Financial Assistance

17 programs

FINANCIAL LANDSCAPE SUMMARY

17

Total programs

14

Open now

7

Foundation grants

1

Copay cards

7

Travel grants

Foundation Grants7

HealthWell Foundation — Fabry disease

HealthWell Foundation

OpenContact for detailsApply ↗

Good Days — Fabry disease

Good Days

OpenContact for detailsApply ↗

Patient Services Inc — Fabry disease

Patient Services Inc

OpenContact for detailsApply ↗

The Assistance Fund — Fabry disease

The Assistance Fund

OpenContact for detailsApply ↗

Patient Advocate Foundation — Fabry disease

Patient Advocate Foundation

OpenContact for detailsApply ↗

PAN Foundation — Fabry disease

PAN Foundation

ClosedContact for detailsApply ↗

NORD Patient Assistance — Fabry disease

NORD Patient Assistance

OpenContact for detailsApply ↗

Copay Assistance1

Galafold

Amicus

OpenContact for detailsApply ↗

Patient Assistance Programs2

ELFABRIO

Chiesi USA, Inc.

OpenContact for detailsApply ↗

Fabrazyme

Genzyme Corporation

OpenContact for detailsApply ↗

Travel Grants7

CareConnectPSS Copay Assistance Program

Sanofi

OpenContact for detailsApply ↗

Fabry Assist

Fabry Support & Information Group (FSIG)

OpenContact for detailsApply ↗

National Kidney Foundation - Prescription Discount and Assistance Resources

National Assistance Foundation

OpenContact for detailsApply ↗

PAN Foundation - Fabry Disease Premium Assistance Fund

PAN Foundation

ClosedUp to $10K/yrApply ↗

PAN Foundation Fabry Disease Premium Assistance

PAN Foundation

OpenContact for detailsApply ↗

The Assistance Fund - Fabry Disease Financial Assistance Program

The Assistance Fund

OpenContact for detailsApply ↗

The Assistance Fund: Fabry Disease

The Assistance Fund

Contact for detailsApply ↗

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Approved Treatments

3 FDA-approved

ELFABRIO

(PEGUNIGALSIDASE ALFA)Orphan drug

Chiesi USA, Inc.

Fabrazyme

(AGALSIDASE BETA)Orphan drug

Genzyme Corporation

Galafold

(MIGALASTAT HYDROCHLORIDE)Orphan drug

Amicus Therapeutics US, LLC

View full treatment details on disease page →

Clinical Trial Landscape

46 active trials

7Phase 3

1Phase 4

1Phase 2

2Phase 1

18N/A

12Unknown

2PHASE1, PHASE2

1EARLY_PHASE1

2PHASE2, PHASE3

46Total recruiting

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Recent News & Research

1 article

NEJMOct 5, 2025

New Oral Treatment for Fabry Disease Shows Promise

A novel oral substrate reduction therapy met its Phase 3 primary endpoint for Fabry disease.

Browse all Fabry disease news →

Specialist Network

Top 6 by expertise

Kate Hanneman

University Health Network

📍 Toronto, Ontario

Score 2622 pubs

Jianhua Mao, PhD

Shanghai Children's Medical Center

📍 Shanghai, Shanghai Municipality

Score 253 trials

eleonora riccio, md

Federico II University

Score 222 trials

Antonio Pisani, MD, PhD

Università degli Studi di Napoli "Federico II" NAPOLI

📍 ROCHESTER, NY

Score 122 trials1 pub

Albert HAGEGE, Dr

20 Rue Leblanc, HEGP/PARCC, 75015 Paris

📍 Paris, Paris

Jeanine R. Jarnes, PharmD

University of Minnesota - Fairview

📍 MINNEAPOLIS, MN

Score 113 trials

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