Clinical TrialsInformation and insight on the latest clinical trials
- ELX-02: Emerging Therapy for Cystic Fibrosis
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company’s leading drug candidate, ELX-02 for cystic fibrosis.
- Alexion Submits BLA For Priority Review And Approval Of ALXN1210
Alexion Pharmaceuticals has submitted a Biologics License Application (BLA) to the YOU.S. Food and Drug Administration (FDA) for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, potentially deadly disease of the blood.T
- First Participant Dosed in Phase 2 Study of Vosoritide for Children with Achondroplasia
BioMarin Pharmaceutical dosed the first participant in their global phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia.
- FDA Grants Orphan Drug Designation to New Treatment for Rett Syndrome
The U.S. Food and Drug Administration has granted Orphan Drug Designation to AMO Pharma for it’s investigational therapy called AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.
- Phase 3 Trials of Rare Genetic Obesity Disorders Completes Enrollment
Rhythm Pharmaceuticals announced that it has completed enrollment of the pivotal cohorts of 10 patients in two separate, ongoing, registration-enabling Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity.
- Patient-Centric Perspective Offers Key Insights into Upcoming Mitochondrial Treatment Studies
As clinical trials gear up with the aim of attaining the first FDA-approved treatments for mitochondrial disease, a new study reports for the first time what patients and families say would motivate them for or against participating in such research trials. Mitochondrial genetic disorders refer to a group of conditions that affect the mitochondria (the structures in each cell of the body that are responsible for making energy).
- 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.
- FDA Approves Combination of Venclexta and Rituxan for Lymphocytic Leukemia
The Food and Drug Administration (FDA) granted approval to venetoclax for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.
- FDA Grants Expedited Status for bluebird bio’s Gene Therapy for CALD
The FDA has granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder.
- Positive Results from Phase 3 OLYMPUS Trial for UTUC
UroGen Pharma presented positive data from an interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel), an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).