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treatment

Iptacopan (LNP023)

Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently in clinical development for treating PNH.

Pegcetacoplan

EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with

Andrew Brunner

Brunner, Andrew
Assistant Professor of Medicine
Harvard Medical School

Dr. Andrew Brunner is an Assistant Professor, Medicine at Harvard Medical School and Assistant in Medicine at the Center for Leukemia at Massachusetts General Hospital in Boston, Massachusetts. His research is focused toward understanding treatment patterns and patient outcomes in MDS and other advanced hematologic malignancies, and to develop new treatments for these patients. He is involved in clinical trials for all stages of MDS with an overall goal of tailoring treatment to improve the outcomes of these patients. 

ASTEX PHARMACEUTICALS PRESENTS OVERALL SURVIVAL DATA FROM ASCERTAIN PHASE 3 STUDY OF ORAL HYPOMETHYLATING AGENT INQOVI® (DECITABINE AND CEDAZURIDINE) IN MDS AND CMML AT INTERNATIONAL CONGRESS ON MYELODYSPLASTIC SYNDROMES

Study achieved median overall survival of 31.7 months
Updated efficacy data demonstrated an overall response rate of 62%, with 22% of patients achieving a complete response
INQOVI is the only oral hypomethylating agent with equivalent exposure to its intravenous (IV) form

sirolimus

A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties.

Eric Yang, MD

Yang, Eric
Associate Clinical Professor of Medicine
Ronald Reagan University of California, Los Angeles Medical Center

Dr. Eric Yang is currently an Associate Clinical Professor of Medicine at the Ronald Reagan University of California, Los Angeles Medical Center. He obtained his undergraduate and medical degrees at the University of Illinois at Chicago College of Medicine, and completed his internal medicine residency and cardiology fellowship at Harbor-UCLA Medical Center, where he served as chief fellow.

"I Chose Life" - Debra Reflects on Three Years of Challenges with Aplastic Anemia

Person's Name: 
Debra O'Neal

“Your blood counts are nearly nonexistent,” the doctor said. 

Before this moment, Debra considered herself to be generally healthy, just tiring easily.  She assumed the cause was the big project at work on top of a host of family and volunteer activities, or perhaps issues with her blood pressure. It was autumn of 2017.

Bone Marrow Disease(s): 
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