BioPharmaFocus on the pharmaceutical and biotech industry
- Stoke Therapeutics: Potential Therapy for Dravet Syndrome
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview the company’s TANGO (Targeted Augmentation of Nuclear Gene Output) technology.
- BioPontis Alliance: Focusing on Rare Disease Drug Development
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s focus on rare diseases, with two programs on Charcot-Marie-Tooth disease and Huntington’s Disease.
- Invitae Discussing Their Presence at the 8th Orphan Drugs and Rare Diseases Conference
Katherine Stueland, Chief Commercial Officer at Invitae, provides an overview of her company and their attendance at this year’s 8th Orphan Drugs and Rare Diseases conference in Boston, MA in September.
- 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.