Clinical Trials

Information and insight on the latest clinical trials
iSCAD Registry

iSCAD Registry

Malissa J. Wood, MD at Harvard Medical School discusses the iSCAD Registry, which develops and maintains an independent, multi-center data repository to advance the pace and breadth of SCAD research around the world. Spontaneous coronary artery dissection (SCAD) is a rare condition and occurs when a tear forms in one of the blood vessels in the heart.

FDA Grants Fast Track Designation for Neurofibromatosis Type 1 Therapy

FDA Grants Fast Track Designation for Neurofibromatosis Type 1 Therapy

The U.S. Food and Drug Administration (FDA) granted Fast Track designation for PD-0325901, an investigational, oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of patients 2 years of age or older with neurofibromatosis type 1-associated inoperable plexiform neurofibromas that are progressing or causing significant morbidity.

Dup15q Research: The ARCADE Trial

Dup15q Research: The ARCADE Trial

Vanessa Vogel-Farley, Executive Director of the Dup15q Alliance, discusses research in this rare disease, including the ARCADE trial, a phase 2 open-label pilot study. 

Usher Syndrome Overview

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.

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