ResearchFocus on the pharmaceutical and biotech industry
Claudio Santos, MD, MBA, of PTC Therapeutics describe the pathophysiology of Aromatic l-amino acid decarboxylase (AADC) deficiency and how it is diagnosed. AADC deficiency is a rare genetic disorder due to mutations in the DDC gene that regulates the AADC...
Adrian C. Newland, MD, of the Royal London Hospital in London, U.K. provided an update on the Phase 2 clinical trial looking at the safety and efficacy of efgartigimod to treat primary immune thrombocytophenia (ITP).
David Roth, MD of Syros Pharmaceuticals describes his company’s gene control methodology to target genetic conditions in a unique way.
Bobby Gasper, MD, Chief Scientific Officer of Orchard Therapeutics, discusses gene therapy being developed to treat children with metachromatic leukodystrophy (MLD).
David Pearce, PhD of Sanford Research discusses how organizations can partner in rare diseases.
Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine talks about the incidence and treatment options for thyroid eye disease due to Graves’ disease.
Femida Gwady-Sridhar, PhD, founder of Pulse Infoframe talks about the need for better collaboration between patient groups and researchers in regard to collecting and using data more effectively and efficiently.
Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc).
The U.S. Food and Drug Administration (FDA) has accepted Genentech’s Biologics License Application (BLA) for satralizumab to treat of adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD).
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics talks about his company’s gene therapy in development to treat children with adenosine deaminase severe combined immunodeficiency (ADA-SCID).