ResearchFocus on the pharmaceutical and biotech industry
- SOBI Overview at ASH: Data from Phase 2/3 Clinical Study of Emapalumab-lzsg in Primary HLH
Rami Levin, President of North America for Swedish Orphan Biovitrum (SOBI) discusses his company’s recent FDA approval, Sobi’s commitment to the rare disease community, and how Sobi is growing its presence in North America.
- Gorlin Syndrome Overview
Jean Tang, MD, PhD is co-founder of PellePharm and serves on the PellePharm board of directors. She is a practicing dermatologist and associate professor of dermatology at Stanford University.
- BioPontis Alliance Overview
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s business model and focus on rare diseases.
- iPATH Study: Key Biomarkers That Affect Bleeding Patterns, Response to Treatment for Hemophilia A
Peter Turecek, Global Medical Affairs, Shire, discusses the Irish Personalised Approach to the Treatment of Haemophilia (iPATH) study, an ongoing collaboration investigating new personalized treatment approaches by tailoring care based on the needs of individual patients.
- Vascular Ehlers-Danlos Syndrome (vEDS)
William T. Andrews, MD, FACP, Chief Medical Officer of Acer Therapeutics discusses his company’s focus on Vascular Ehlers-Danlos Syndrome (vEDS), an inherited connective tissue disorder that is caused by defects in a protein called collagen.
- FDA Approves First Treatment for Lambert-Eaton Myasthenic Syndrome
Some long-awaited good news arrived for the Lambert-Eaton Myasthenic syndrome (LEMS) community this week as the U.S. Food and Drug Administration approved Firdapse (amifampridine) tablets for the treatment of LEMS in adults.
- Partnering With BioPontis Alliance
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s business model, focus on rare diseases, and willingness to partner with patients, academic centers, investors, and volunteers.
- 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.