Clinical TrialsInformation and insight on the latest clinical trials
- Using Genomics to Better Understand Rare Diseases
Daniel MacArthur, PhD is an Institute Member at the Broad Institute of MIT and Harvard, and co-lead of the Broad’s Center for Mendelian Genomics and Rare Genomes Project.
- FDA Approves Hemlibra for Hemophilia A Without Factor VIII Inhibitors
The FDA expanded the approval of Hemlibra (emicizumab-kxwh) to include routine prophylaxis to prevent or reduce the frequency of bleeding episodes among individuals with hemophilia A without factor VIII inhibitors.
- Translational Science at the NIH: Moving Beyond “One Disease at a Time”
Philip John Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health (NIH) discusses his work in rare diseases at the NIH.
- Sarepta Presents Positive Gene Therapy Data for Duchenne Muscular Dystrophy
Sarepta Therapeutics announced that at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, MD, of Nationwide Children’s Hospital presented positive updated results from its gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy (DMD).
- 22q11.2 Deletion Syndrome
Bernice Morrow, PhD, Director of Translational Genetics, Albert Einstein College of Medicine, talks about the challenges of developing treatments for 22q11.2 deletion syndrome, a disorder caused by the deletion of a small piece of chromosome 22.
- 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.