by Peter Ciszewski | Dec 11, 2017
For people with certain types of aggressive, refractory blood cancers, treatment options are limited. But three studies being presented at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta spotlight the emerging role played by...
by Peter Ciszewski | Dec 8, 2017
The rare disease industry – including patients, researchers, and biotech companies – are concerned that a key financial provision in the Orphan Drug Act of 1983 may soon be removed. These financial incentives have been key driver for biotech companies to pursue...
by Peter Ciszewski | Dec 8, 2017
Spark Therapeutics and Pfizer announced that The New England Journal of Medicine has published interim data from the Phase 1/2 clinical trial of SPK-9001, an investigational gene therapy for hemophilia B. With a cumulative follow-up of 492 weeks’ observation of the...
by Peter Ciszewski | Dec 7, 2017
David Gelly is a patient with Polycythemia Vera (PV). In this interview he discusses some of the resources available to patients with this rare condition, including Voices of MPN. PVs are a group of rare blood cancers called Myeloproliferative Neoplasms (MPNs). MPNs...
by Peter Ciszewski | Dec 6, 2017
David G. Birch, PhD, Chief Scientific and Executive Officer and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, Retina Foundation of the Southwest discusses genetic testing and X-linked retinoschisis (XLRS). X-linked juvenile retinoschisis is a...
