by Peter Ciszewski | Mar 4, 2019
Kyle Bryant is a rare disease advocate. In this video, Kyle discusses Friedreich’s Ataxia (the rare disease he lives with) and Rare Disease Day. Kyle was diagnosed with Friedreich’s Ataxia at the age of 17. Friedreich ataxia is a rare inherited disease that...
by Peter Ciszewski | Mar 3, 2019
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s pipeline which is focused on a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and...
by Peter Ciszewski | Mar 1, 2019
Armando Anido, Chief Executive Officer at Zynerba Pharmaceuticals discusses Autism Spectrum Disorder (ASD), a developmental disorder that affects communication and behavior. Zynerba is pursuing new therapeutic options for ASD and other neuropsychiatric...
by Peter Ciszewski | Feb 28, 2019
Rare Disease Day is a day for those in the rare disease community to share their stories. There are over 7000 rare diseases and each person who has a rare disease, or who is working to find a treatment for a rare disease, has a unique story to tell. CheckRare, an...
by Peter Ciszewski | Feb 28, 2019
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s proprietary chimeric antigen receptor engineered T cell (CAR T) immunotherapies program. Mustang Bio is focused on the development of a broad range of...
