ResearchFocus on the pharmaceutical and biotech industry
- Stoke Therapeutics: Focus on Rare Genetic Diseases
Barry Ticho is the Chief Medical Officer at Stoke Therapeutics, and in this clip he provides an overview of his company.
- FDA Approves Clobazam Oral Film (Sympazan) for Lennox-Gastaut Syndrome
The US Food and Drug Administration (FDA) approved an oral film formulation of clobazam (Sympazan, Aquestive Therapeutics) for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients 2 years and older.
- How Diagnostic Testing Companies Work with Biotech
Katherine Stueland, Chief Commercial Officer at Invitae, discusses how diagnostic companies work with biotech companies. Katherine also discusses the role of genetic companies in assisting patients and physicians in diagnosing rare diseases.
- The Role of Advocacy Groups in Cystic Fibrosis Drug Development
Greg Duncan, President and Chief Executive Officer of Celtaxsys, discusses working with advoacy groups such as the Cystic Fibrosis Foundation, on their clinical trials.
- The Growth of the Biotech Industry in Cambridge, MA
Barry Greene, President of Alnylam, discusses the growth of the biotech industry in Cambridge. MA.
- Celtaxsys: Focus on Cystic Fibrosis and Rare Inflammatory Diseases
Greg Duncan, President and Chief Executive Officer of Celtaxsys, provides an overview of his company and it’s focus on cystic fibrosis (CF), an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide.
- How Alnylam Works with Patient Advocacy Groups
Barry Greene, President of Alnylam, discusses how his company works with advocacy groups in the diseases they are committed to.
- Jeremy Levin: Challenges of Working with Rare Diseases
Jeremy Levin, Chairman and Chief Executive Officer of Ovid Therapeutics, discusses the challenges of a small biotech company working with rare diseases, especially rare neurological disorders.
- Albiero Pharma Overview and Focus on Rare Pediatric Liver Diseases
Ron Cooper, President and CEO of Albireo, discusses his company and lead product. Albireo is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat orphan pediatric liver diseases and other liver or gastrointestinal diseases and disorders.
- Focus on Cystinosis
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of Cystinosis, an inherited condition in which the body accumulates the amino acid cystine (a building block of proteins) within the cells.