Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical Trials and Children

Until recently, children were rarely included in studies of medical treatments. As a result, much is still unknown about how children respond to drugs, some biologics (such as gene therapy) and medical devices. According to the U.S. Food and Drug Administration (FDA), only 20-30% of approved drugs have actually been labeled for use in children.

mRNA COVID-19 Vaccination Sparks Strong Antibody Response in Patients With AML and MDS

Original Publication Date
Article Source
External Web Content
The mRNA COVID-19 Vaccines Are Safe and Effective for Most Patients with Cancer For most patients with cancer, the mRNA COVID-19 vaccines are safe and effective. This article explains the results of the first study that evaluated the safety and effectiveness of the mRNA vaccines…

Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH

Original Publication Date
Article Source
External Web Content
Published: Dec 15, 2021 STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- The first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1 Aspaveli will…

BCX9930

is an oral small molecule inhibitor

Taha Bat, MD

Institution
UT Southwestern Medical Center
Physician Status
accepting new patients
Primary Disease Area of Focus
Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eloy Roman, MD

Institution
Lakes Research
Physician Status
accepting new patients
Primary Disease Area of Focus
Acute Myeloid Leukemia (AML)
Aplastic Anemia
Myelodysplastic Syndromes (MDS)
Myeloproliferative Neoplasms (MPN)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
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