Resources for Patients Suffering from Polycythemia Vera

David Gelly, a patient with Polycythemia Vera (PV), discusses some of the resources available for this rare condition, including Voices of MPN. PVs are a group of rare blood cancers called Myeloproliferative Neoplasms (MPNs). MPNs are a group of diseases that affect...

Pulmonary Arterial Hypertension Overview

Roxana Sulica, MD, Associate Professor, Department of Medicine and Director, Pulmonary Hypertension at NYU Langone in New York City discusses Pulmonary arterial hypertension (PAH), a progressive condition that affects the heart and lungs. PAH is characterized by...

A Look Back at Some of the Emerging Biotech Successes in 2017

Sam Falsetti, PhD of Cambridge Biomarketing discusses some of the biotech and rare disease successes of 2017, including the emergence of RNA therapies with the approval of Exondys 51 for Duchenne muscular dystrophy, as well as gene and CAR-T therapies that have had...

Emerging Treatments for Desmoid Tumors

Lara Sullivan, MD, President and Founder at SpringWorks Therapeutics discusses desmoid tumors, a rare, non-metastatic tumor of connective tissue cells, which can cause severe morbidity, pain and loss of function in children and adults. Desmoid tumors can show up in...

The MOA of Senicapoc: A Gardos Channel Blocker

Lara Sullivan, MD, President and Founder at SpringWorks Therapeutics discusses the mechanism of action of Senicapoc (a Gardos channel blocker), an emerging treatment for Hereditary Xerocytosis, a genetic disorder in which red blood cells become dehydrated due to loss...
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