Rare Disease Report


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.
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, M.D., Director at the Hemotosis and Thrombosis Center at CHLA thinks that we're in the middle of a revolution for hemophilia treatment.
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 Pharmaceuticals President and CEO Raul Rodriguez is encouraged by his company's success and the FDA's recent activity focused on rare hematologic disease.

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.
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.
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.
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.
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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