Clinical TrialsInformation and insight on the latest clinical trials
- 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.
- Positive Topline Results from Phase 3 Trial of Charcot-Marie-Tooth Type 1A Disease
Pharnext announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A).
- FDA Grants Fast Track Designation to A4250 for Alagille Syndrome
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome, a rare and life-threatening disease that affects the liver and has no approved pharmacologic treatment option.
- Dravet Syndrome Overview
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview of Dravet syndrome, a severe form of epilepsy that is part of a group of diseases known as SCN1A-related seizure disorders.