Overview of Chronic Neutropenic Disorders

Julia Warren, MD, Ph.D., Associate Professor of Hematology at the University of Pennsylvania and Hematologist at Children’s Hospital of Philadelphia, gives an overview of chronic neutropenic disorders.   Chronic neutropenic disorders are blood disorders...

Bernie Williams: Tune in To Lung Health

  Baseball legend, Bernie Williams, formerly of the  New York Yankee (and currently a jazz musician), discusses his advocacy work to raise awareness of interstitial lung diseases, including idiopathic pulmonary fibrosis (IPF).  There are IPF is a chronic,...

HoFH Patient Diagnostic Journey

  An HoFH patient describes his diagnostic journey following years of symptoms. Peter, a resident of San Diego, was diagnosed with homozygous familial hypercholesterolemia  (HoFH) at age 27 by an alert urgent care physician. In hindsight, Peter had displayed...

Treatment of Charcot-Marie-Tooth Disease Type 1A

Arthur Suckow, PhD., chief executive officer at DTx Pharma talks about Charcot-Marie-Tooth Disease Type 1A (CMT1A) and a treatment in development for this rare disease. CMT1A is a rare inherited neurological disorder that causes damage to the peripheral nerves. It is...

Treatment Testing for Myotonic Dystrophy Type 1 (DM1) 

  Art Levin, PhD, distinguished scientist and strategic leader and member of the company’s board of directors at Avidity Biosciences, discusses positive data from the preliminary assessment of the phase 1/2 MARINA trial of AOC 1001 for treatment in patients with...