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BioWorld - Saturday, February 14, 2026
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Phase I data on SLN-124 support ongoing evaluation in patients with thalassemia and MDS

May 19, 2021

Anti-BAFF therapy eradicates factor VIII inhibitors in hemophilia A

May 19, 2021

First patient dosed in phase I/II MAA-202 study of MarzAA

May 19, 2021
Empaveli

An alternative to C5 inhibitors, Apellis’ pegcetacoplan wins FDA approval in PNH

May 17, 2021
By Lee Landenberger
Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.
Read More

FDA approves Empaveli for PNH

May 17, 2021

FDA approves Ferriprox to treat transfusional iron overload due to sickle cell disease

May 3, 2021

European Commission approves Orladeyo for the prevention of HAE attacks

May 3, 2021

Hillhurst cleared by FDA to begin phase I study of HBI-002 for sickle cell disease

April 28, 2021

FDA removes clinical hold on uniQure's hemophilia B gene therapy program

April 27, 2021

Fulcrum Therapeutics' FTX-6058 could be a disease-modifying treatment for sickle cell disease

April 26, 2021
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