ResearchFocus on the pharmaceutical and biotech industry
Jeff Ajer, Executive Vice President and Chief Commercial Officer of BioMarin, discusses gene therapy. The development of these transformative therapies has triggered discussions about market access challenges, the viability of alternative financing mechanisms, and the results for patient access.
Raj Mehra, PhD, Chief Executive Officer of Seelos Therapeutics provides an overview of Sanfilippo syndrome, also known as Mucopolysaccharidosis type III (MPS III), a progressive disorder that primarily affects the brain and spinal cord (central nervous system).
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).
Stefan Weber, CEO of Newron Pharmaceuticals, provides an update on the “Sarizotan Treatment of Apneas in Rett Syndrome” (STARS) study, a potential pivotal clinical study performed in study centers in the US, Europe, Asia, and Australia to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms.
Hartmann Wellhoefer, MD, Vice President, Medical Affairs, Rare Disease and Internal Medicine, Global R&D at Takeda sat down with CheckRare to discuss the 15th Annual WORLDSymposium 2019 to share his insights about this year’s conference including key highlights and trends and Takeda’s presence.
Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses Rare Disease Day and his organization which is one of the largest health systems in the United States, including research into rare diseases.
Monica Weldon of Bridge The GAP – SYNGAP talks about this rare neurological condition that is linked to autism.
Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses his organization which is one of the largest health systems in the United States, including research into rare diseases.
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses the KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma.
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses gene testing for inherited eye diseases, including his company’s initative EyeWant2Know.com.