Clinical TrialsInformation and insight on the latest clinical trials
- Usher Syndrome Overview
Daniel de Boer, Founder and CEO of ProQR, discussuses Usher syndrome, a genetic disorder characterized by sensorineural hearing loss or deafness and progressive vision loss due to retinitis pigmentosa.
- A Novel Protein Therapy in Development for Achondroplasia (Short-Limbed Dwarfism)
Luca Santarelli, MD of Therachon discusses his company’s lead pipeline candidate, TA-46, a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism.
- FDA Allows Use of Kalydeco in Children Under Two Years Old
The U.S. Food and Drug Administration (FDA) has approved ivacaftor (KALYDECO) to be used in children with cystic fibrosis ages under the age of 2 years old who have at least 1 mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the drug.
- Positive Results in Phase 3 Cushing’s Syndrome Trial
Strongbridge Biopharma’s pivotal phase 3 SONICS study evaluating levoketoconazole (Recorlev) for the treatment of endogenous Cushing’s syndrome achieved statistical significance of its pre-specified primary endpoint.
- Correlation Between NIHF and MPS VII
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses the correlation between non-immune Hydrops fetalis (NIHF) and MPS VII.
- Alnylam’s New RNAi Therapy
Barry Greene, President of Alnylam, provides RNA interference (RNAi) and the first ever new class of innovative medicines to potentially enter the market this year.
- New License Agreement for the Development of Trofinetide in Rett Syndrome
Acadia Pharmaceuticals and Neuren Pharmaceuticals announced that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications.
- Progressive Familial Intrahepatic Cholestasis (PFIC) Clinical Trials
Ron Cooper, President and CEO of Albireo, provides and overview of progressive familial intrahepatic cholestasis type (PFIC) is a rare condition that affects the liver.
- The Right to Try Act
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses Right To Try, which is aimed at allowing terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves.
- Personalized Medical Therapies for Emerging Food Allergy Disorder
Scientists at Cincinnati Children’s Hospital Medical Center have uncovered three distinct subtypes of eosinophilic esophagitis (EoE), an emerging food allergic disease which may provide a framework for developing precision medicines to treat this often-debilitating disorder.