Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs
The current standard of care for
paroxysmal nocturnal hemoglobinuria
paroxysmal nocturnal hemoglob
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Clinical development of our investigational therapy, pegcetacoplan
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Dear members of the PNH community, We are excited to share important updates with you regarding the clinical development of our investigational therapy, pegcetacoplan, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Since our last update, we have partnered with…
Pegcetacoplan Bests Eculizumab for PNH Patients
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Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy…
Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure
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Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare, acquired, life-threatening disease of the blood. The name comes from one of the clinical consequences of the disease -- the breakdown of red blood cells and consequent release of hemoglobin into the urine. It often…
Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease
In the UK, early work on paroxysmal nocturnal haemoglobinuria (PNH) was conducted by John Dacie who, at the Hammersmith Hospital, first hypothesised that the PNH abnormality might arise through a somatic mutation mutation:
Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (pa
PNH - Traveling with a Rare Disease - Update from Josh
Traveling with a rare disease
Crovalimab
An experimental complement inhibitor C5 monoclonal antibody monoclonal antibody: A type of protein called an antibody that is engineered to look for a speci
David Jacobsohn, MD, ScM
Institution
Children's National
Physician Status
accepting new patients
Primary Disease Area of Focus
Acute Myeloid Leukemia (AML)
Aplastic Anemia
Chronic Lymphocytic Leukemia (CLL)
Chronic Myelomonocytic Leukaemia (CMML)
Graft Versus Host Disease (GVHD)
Myelodysplastic Syndromes (MDS)
Myeloproliferative Neoplasms (MPN)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia (PRCA)
Pediatric
About
David Jacobsohn, MD, ScM is the Division Chief, Blood and Marrow Transplanation at Children's National Hospital in Washington, D.C. Previously, he was at Children’s Memorial Hospital in Chicago, where he served as a member of the Stem Cell Transplant Program staff and as the Director of the Chronic Graft-versus-Host Disease Clinic. He has lectured widely on GvHD/BMT in Europe, Latin America, and the United States. Dr. Jacobsohn obtained a Bachelor of Science from Yale University, his medical degree from Tufts University, and a Masters in Clinical Investigation from Johns Hopkins School of