Advocacy intelligence hub — real-time data for patient organizations
Novartis Pharmaceuticals
Nanjing Chia-tai Tianqing Pharmaceutical — PHASE3
NovelMed Therapeutics — PHASE2
NovelMed Therapeutics — PHASE2
Regeneron Pharmaceuticals — PHASE3
Shenzhen Kangzhe Biotechnology Co., Ltd. — PHASE1
Jiangsu Hansoh Pharmaceutical Co., Ltd. — PHASE1, PHASE2
Regeneron Pharmaceuticals — PHASE1, PHASE2
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Open now
Foundation grants
Copay cards
Travel grants
Foundation Grants2
Copay Assistance7
BKEMV
Amgen
FABHALTA
Novartis
Empaveli
Apellis
Voydeya
Alexion
SOLIRIS
Alexion
Ultomiris
Alexion
Piasky
Genentech
Travel Grants11
Alexion Access Navigator - SOLIRIS Access and Reimbursement Guide
Alexion
Alexion OneSource - Navigating Insurance for SOLIRIS Patient Guide
Alexion OneSource
Alexion OneSource program (via National Kidney Foundation)
PAN Foundation - Paroxysmal Nocturnal Hemoglobinuria Fund
PAN Foundation
PAN Foundation Paroxysmal Nocturnal Hemoglobinuria Financial Assistance
PAN Foundation
PAN Foundation Paroxysmal Nocturnal Hemoglobinuria Fund
PAN Foundation
Paroxysmal Nocturnal Hemoglobinuria Financial Assistance Program
The Assistance Fund
PNH Patient Travel Assistance Fund
Aplastic Anemia and MDS International Foundation (AAMDSIF)
The Assistance Fund (TAF) - Paroxysmal Nocturnal Hemoglobinuria Financial Assistance
The Assistance Fund (TAF)
The Assistance Fund Paroxysmal Nocturnal Hemoglobinuria Financial Assistance Program
The Assistance Fund
ULTOMIRIS Savings and Affordability
FABHALTA
(IPTACOPAN)Orphan drugNovartis Pharmaceuticals Corporation
BKEMV
(ECULIZUMAB-AEEB)Orphan drugAmgen Inc
Ultomiris
(RAVULIZUMAB)Orphan drugAlexion Pharmaceuticals Inc.
Voydeya
(DANICOPAN)Orphan drugAlexion Pharmaceuticals Inc.
SOLIRIS
(ECULIZUMAB)Orphan drugAlexion Pharmaceuticals Inc.
EPYSQLI
(ECULIZUMAB-AAGH)Orphan drugTeva Pharmaceuticals USA, Inc.
Piasky
(CROVALIMAB)Orphan drugGenentech, Inc.
Empaveli
(PEGCETACOPLAN)Orphan drugApellis Pharmaceuticals, Inc.
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-m...
Complement blockade: Therapeutic promises and remaining challenges in clinical practice.
Understanding of the complement system in glomerulopathies has advanced significantly, revealing that this system-beyond its role in innate immunity-is a key mediator of kidney injury across a wide sp...
Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired stem-cell disease causing anemia, aplasia and thromboses, affecting ~1/80,000 patients in France. Treatment mainly involves C5 inhibitors ...
Ravulizumab for Treatment of Paroxysmal Nocturnal Hemoglobinuria During Pregnancy.
Ravulizumab, a humanized, long-acting monoclonal antibody against complement factor C5, is a widely utilized treatment for paroxysmal nocturnal hemoglobinuria (PNH). As pregnant women with PNH are at ...
Acute Pancreatitis as a Therapeutic Complication in Case of Paroxysmal Nocturnal Hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic disorder requiring careful therapeutic management. In resource-limited settings, traditional treatments may carry significant...
Case report: persistent anemia after eculizumab in paroxysmal nocturnal hemoglobinuria: non-dominantly active intravascular hemolysis.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by intravascular hemolysis (IVH), thrombosis, and organ damage. Although C5 complement inhibitors such as eculizumab have si...
Steve Whitaker, MD
Omeros Corporation
Camille Bedrosian, MD, MD
Amylyx Pharmaceuticals
📍 CAMBRIDGE, MA
Nader Najafian, MD, MD
Alnylam Pharmaceuticals
📍 BOSTON, MA
Brandon Triplett, MD, MD
St. Jude Children's Research Hospital
📍 MEMPHIS, TN
Luciano A Sposato, MD
London Health Sciences Center, Western University
📍 London, Ontario
Ashish Gupta, MBBS, MPH
Masonic Cancer Center, University of Minnesota