Clinical TrialsInformation and insight on the latest clinical trials
- Results of Phase 2 Trial Showing Improvements in Pulmonary Exacerbations in Cystic Fibrosis
Greg Duncan, CEO of Celtaxsys discusses the top line results of its Phase 2 EMPIRE-CF trial evaluating oral, once daily anti-inflammatory molecule, acebilustat, for the treatment of cystic fibrosis (CF), irrespective of the causative genotype.
- Hemophagocytic Lymphohistiocytosis: Disease Overview and Emerging Treatment Options
en Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and lymphocytes).
- NIH to Accelerate Genetic Therapies for Sickle Cell Disease
The National Institutes of Health (NIH) today announced the launch of a new initiative to help speed the development of cures for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide.
- FDA Grants Fast Track Designation to Bullous Pemphigoid Treatment
The U.S. Food and Drug Administration (FDA) granted Fast Track designation to bertilimumab for the treatment of bullous pemphigoid, a skin disorder characterized by large blisters.
- 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.
- Angelman Syndrome Overview
Amit Rakhit, MD, Chief Medical and Portfolio Officer at Ovid Therapeutics, provides an overview on Angelman syndrome. Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. Characteristic features of this condition i …
- FDA Approves Tiglutik for the Treatment of Amyotrophic Lateral Sclerosis
The US Food and Drug Administration (FDA) approved Tiglutik (riluzole) oral suspension for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
- Buergers Disease Treatment Receives Orphan Drug Designation
U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATI-1013, a fully human, anti-nicotine monoclonal antibody for the treatment of Buerger’s Disease.
- Novel Therapy for Cystic Fibrosis Completes Phase 2 Trial
Greg Duncan, President and Chief Executive Officer of Celtaxsys, provides an overview of cystic fibrosis (CF), an inherited chronic disease that affects the lungs and digestive system of about 70,000 children and adults worldwide.
- ATTR Study Shows Tafamidis Minimizes Mortality and Hospital Visitation
Pfizer announced positive data from it’s phase 3 study (called ATTR-ACT), which evaluated it’s experimental therapy Tafamidis (vyndaqel) in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM).