ResearchFocus on the pharmaceutical and biotech industry
- Catalyst Biosciences: Developing Novel Medicines to Address Hematology Indications
Nassim Usman, PhD, President and Chief Executive Officer of Catalyst Biosciences provides an overview of his company and its focus on protease-based therapeutic candidates.
- Acute Hepatic Porphyria: Overview and Emerging Therapies
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Acute Hepatic Porphyria (AHPs) and givosiran, an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP.
- Hereditary ATTR Amyloidosis Overview
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses hereditary ATTR (hATTR) amyloidosis, a rare condition that affects an estimated 50,000 people worldwide.
- Positive Top-Line Results From Phase 3 Study Of Ultomiris in Atypical Hemolytic Uremic Syndrome
Alexion announced positive top line results in the Phase 3 study of Ultomiris (ravulizumab-cwvz), the long-acting C5 complement inhibitor, met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (aHUS).
- Proteus Syndrome: Overview and Emerging Therapies
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company focus on Proteus syndrome, a rare condition characterized by overgrowth of the bones, skin, and other tissues.
- Potential Use of Venetoclax in Multiple Myeloma
Rod Humerickhouse, MD, PhD, asset strategy leader at AbbVie discusses the potential use of venetoclax for the treatment of patients with multiple myeloma at the 60th ASH Annual Meeting in San Diego.
- Primary Haemophagocytic Lymphohistiocytosis: Overview and New Treatment Option
Carol Satler, MD Vice President of Medical Affairs in North America for Sobi, discusses the ultra rare disease primary haemophagocytic lymphohistiocytosis (HLH) and a new FDA approved treatment Emapalumab.
- 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.