Emmaus Presents Endari Data at ASH
Rare Disease Report sat down with Yutaka Niihara, M.D., MPH, to discuss the clinical trials for Endari, and when the drug will be available to patients.
The Evolving Paradigm in Treatment for Hodgkin Lymphoma
At the 59th ASH Annual Meeting and Exposition,
Rare Disease Report
sat down with Clay Siegall to discuss why the plenary data presented over the weekend was so important to the rare cancer community.
Guy Young, MD, on the Recent Revolutions in Hemophilia Treatment
Guy Young, M.D., Director at the Hemotosis and Thrombosis Center at CHLA thinks that we're in the middle of a revolution for hemophilia treatment.
MPN Expert Ruben Mesa on the Anemia-Based Data at ASH
UT Health Cancer Center Director Ruben Mesa, MD, explains the anemia that can sometimes come with MPN-associated myelofibrosis, and the pertinent data presented at ASH 2017.
Rigel CEO Encouraged by Company's Progress and Recent FDA Activity
Rigel Pharmaceuticals President and CEO Raul Rodriguez is encouraged by his company's success and the FDA's recent activity focused on rare hematologic disease.
CMO of Catalyst Biosciences Explains the Current State of Hemophilia
In this video, Howard Levy, , MBBCh, PhD, MMM, CMO of Catalyst Biosciences believes this is a promising time for the hemophilia community, and while gene therapy can be exciting, that conventional therapies are still the way to go.
Oncology Expert Explains Why These Are Exciting Times in Rare Cancers
Gerald Messerschmidt, M.D., FACP, has worked in oncology for 37 years. Watch this video and find out why he thinks we're living in exciting times as it pertains to rare cancer research.
Is Soliris Effective in aHUS with Complement-Amplifying Conditions?
Julia M Cunningham, MD presented results for 52 aHUS patients who received Soliris (eculiuzmab) between December 2011 and February 2017 to assess the drug’s impact.
Eryaspase Fails to Meet Primary Endpoints in AML Study
It was announced this morning that results from a Phase 2b study conducted by ERYTECH Pharma that eryaspase showed an inability to meet its overall survival endpoint in AML.
NEJM Publishes Data from Study of Gene Therapy in Hemophilia B
The New England Journal of Medicine
has published interim data from Spark Therapeutics' Phase 1/2 clinical trial of SPK-9001 in patients with hemophilia B.
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