Clinical TrialsInformation and insight on the latest clinical trials
- Luspatercept Successful in Late-Stage Beta Thalassemia Study
A Phase 3 clinical trial evaluating luspatercept in adults with beta-thalassemia-associated anemia met the primary endpoint. The results were presented at the American Society of Hematology (ASH) in San Diego.
- Krabbe Disease
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Krabbe disease (also called globoid cell leukodystrophy), a degenerative disorder that affects the nervous system.
- Leber’s Congenital Amaurosis Overview
Daniel de Boer, Founder and CEO of ProQR, discussuses Leber’s congenital amaurosis (LCA), the most common genetic cause of childhood blindness.
- FDA Grants Orphan Drug Designation to Gene Therapy for CLN2 Form Of Batten Disease
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.
- Rett Syndrome Natural History Study
Daniel Glaze, Medical Diorector of the Blue Bird Circle Rett Center and a Professor in the Departs of Pediatrics and Neurology at Baylor College of Medicine in Houston discusses the Rett Syndrome Natural History Study.
- Rett Syndrome Research and Clinical Trials
Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departments of Pediatrics and Neurology at Baylor College of Medicine in Houston, discusses ongoing Rett syndrome research and clinical trials, including the ongoing phase 3 STARS trial.
- Sickle Cell Trait Increases Risk for Some Health Conditions
In the United States, an estimated 2.5 to 3 million people have a gene variant associated sickle cell trait (SCT) and most never have related health complications. Researchers have now identified three health conditions for which people with SCT are at increased risk.
- Hemophagocytic Lymphohistiocytosis: Advocacy Groups and Clinical Trials
Len Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses his company’s activities in Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and lymphocytes).
- 10 Things to Know About Clinical Trials
Clinical trials have led to important medical discoveries like vaccines and treatments for cancer, heart disease, diabetes, and rare diseases. Without clinical trials, we wouldn’t have any of the medicines we take every single day. Knowing more about c …
- AACAP Conference Update: Phase 2 STARS Trial for Angelman Syndrome
At this year’s American Academy of Child and Adolescent Psychiatry (AACAP) Annual Meeting in Seattle, Washington, October 22-27, Ovid Therapeutics announced additional efficacy data from the company’s Phase 2 STARS trial that further support the potential of OV101, a novel selective extrasynaptic GABAA receptor agonist that is being investigated to treat Angelman syndrome.