Clinical TrialsInformation and insight on the latest clinical trials
- 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.
- FDA Grants Lynparza Orphan Drug Designation for Pancreatic Cancer
The FDA granted Olaparib (Lynparza) an orphan drug designation by for the treatment of patients with pancreatic cancer,
- Phase 1/2 Study Evaluating SOBI003 for Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA)
Len Walt, Vice President, Medical Affairs, North America discusses his company’s focus on rare diseases, specificially MPS IIIA or Sanfilippo A syndrome.
- FDA Approval of Revcovi for Treating ADA-SCID Patients
The FDA approved Revcovi (elapegademase-lvlr) injection in the United States, for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
- 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.