by Peter Ciszewski | Jul 23, 2019
Recently, we talked with Akshay Vaishnaw, MD, PhD, is Chief Medical Officer at Alnylam Pharmaceuticals about their RNA interference (RNAi) drug, givosiran, that targets aminolevulinic acid synthase 1 (ALAS1) to treat acute hepatic porphyria. Acute hepatic...
by Peter Ciszewski | Jul 19, 2019
John Maslowski, President and Chief Executive Officer (CEO) of Fibrocell Science understands the importance of working with patient advocacy groups to develop rare disease treatments. Recently, we talked to Maslowski about his biotech company and its reliance...
by Peter Ciszewski | Jul 18, 2019
Rare in Common, a podcast hosted by Andra Stratton, regularly posts conversations with leaders in the rare disease community to help them share their stories. The podcast began in 2017 at Cambridge Biomarketing, as an offshoot of the company’s documentary ‘Rare...
by Peter Ciszewski | Jul 17, 2019
The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARO-ANG3 (Arrowhead Pharmaceuticals) for the treatment of homozygous familial hypercholesterolemia (HoFH). ARO-ANG3 is a RNA interference (RNAi)-based medicine that targets angiopoietin...
by Peter Ciszewski | Jul 17, 2019
Odylia Therapeutics is a non-profit corporation working to help develop gene therapies for rare eye disease. Recently, we talked to Harrison Brown, PhD, BBA, the company’s Chief Science Officer about advances in gene therapy. As Dr. Brown explains in this...
