Morie Gertz, MD, Hematologist and Chair of Internal Medicine at Mayo Clinic discussed the current unmet needs in the treatment of hereditary TTR amyloidosis, antisense technology, inotersen, and the NEURO-TTR study.
Annette Bakker, President and Chief Scientific Officer of the Children’s Tumor Foundation, discusses neurofibromatosis (NF), a genetic disorder that affects 1 in every 3,000 people.
The U.S. Food and Drug Administration (FDA) granted PRM-151, a novel investigational anti-fibrotic immunomodulator, Breakthrough Therapy designation for Idiopathic Pulmonary Fibrosis (IPF).
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses the KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma.
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses his company and it’s focus on developing cytokine-based intratumoral immunotherapies to stimulate the body’s immune system to target and attack cancer.
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses biallelic RPE65 mutation-associated retinal dystrophy, a rare retinal disorder.