Advancing Research Through the Global PNH Registry: Q & A with Dr. David J. Araten
The is a pre-recorded session with the original PNH Registry Launch video and audience Q & A.
patient
Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)
– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –
– Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –
Living with GVHD: A Transplant Patient Panel Discussion
Living with PNH: A Patient Panel Discussion
Apellis and Sobi Report Positive Top-line Results from the Phase 3 PRINCE Study of EMPAVELI™ (pegcetacoplan) in Treatment-naïve Patients with PNH
May 25, 2021
Clinical effects of administering leukemia-specific donor T cells to patients with AML/MDS after allogeneic transplant
Key Points
Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo.
Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects.
Researchers Uncover How MN1 Overexpression Causes AML
Venetoclax
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines to treat these conditions.
Aplastic Anemia and MDS International Foundation and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Global PNH Patient Registry
The Global PNH Patient Registry is a collaborative effort between the Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD®) to study PNH.