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Mashup Score: 1
The first-in-class oral factor B inhibitor iptacopan improves hematologic and clinical outcomes in anti-C5-treated patients with persistent anemia and in those who have not received complement inhibitors.
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Mashup Score: 2
For older patients with acute myeloid leukemia undergoing allo-HCT, posttransplant outcomes have improved over time.
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Mashup Score: 0
Exclusive breastfeeding duration of at least three months may decrease the risk for childhood hematologic cancers
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Mashup Score: 02024 Orphan Drugs: PDUFA Dates and FDA Approvals - 7 day(s) ago
Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2022.
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Mashup Score: 5ALS Conversations and Ongoing Platform Trial - 7 day(s) ago
Benjamin Barnes, MD, Augusta University, discusses how he approaches ALS patient conversations and an ongoing platform trial.
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Mashup Score: 1
Sean Studer, MD, MSc, FCCP, discusses the changing treatment landscape of PAH, including the approval of Opsynvi.
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Mashup Score: 1History of Rett Syndrome - 7 day(s) ago
Alan Percy, MD, Pediatric Neurologist at University of Alabama at Birmingham, describes the history of Rett syndrome.
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Mashup Score: 0Tenosynovial Giant Cell Tumor (TGCT) - 7 day(s) ago
Tenosynovial giant cell tumor (TGCT) is a rare benign tumor involving the joint synovium, bursae, and tendon sheath.
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Mashup Score: 1Pridopidine Clinical Trials for ALS and Huntington’s Disease - 7 day(s) ago
Michael Hayden, PhD, CEO of Prilenia, discusses recent clinical trials testing pridopidine for the treatment of ALS and Huntington’s disease.
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Mashup Score: 0
Patients with primary cutaneous B-cell lymphomas (PCBCLs) have an increased risk for second primary malignancies (SPMs).
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The first-in-class oral factor B inhibitor iptacopan improves hematologic and clinical outcomes in anti-C5-treated patients with persistent anemia and in those who have not received complement inhibitors. #CheckRare #RareDisease #Anemia https://t.co/zz9oGFZ5eX