Clinical TrialsInformation and insight on the latest clinical trials
- FDA Grants Orphan Drug Designation to ARO-ANG3 for HoFH
The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARO-ANG3 (Arrowhead Pharmaceuticals) for the treatment of homozygous familial hypercholesterolemia (HoFH).
- Barriers to Recruiting Patients for Rare Disease Clinical Trials
Dennis Jackman, senior vice-president of public affairs at CSL Behring discusses some of the barriers to recruiting participants to be in clinical trials for rare diseases.
- The FDA Grants Orphan Drug Designation LM-030 For Netherton Syndrome
The Food and Drug Administration (FDA) granted orphan drug designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome.
- FDA Grants Orphan Drug Status to Hunter Syndrome Candidate
The FDA has granted orphan drug status and a rare pediatric disease designation to Denali Therapeutics pipeline candidate, DNL310.
- 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.
- Rising to the Challenge: Developing New Medicines for Rare Disease
The process for researching and developing new medicines is notoriously time-consuming, complicated and costly – and even more so when it comes to therapies for rare diseases.
- 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
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.
- Mucopolysaccharidosis Type II (MPS II), Hunter Syndrome
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone Health provides an overview of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
- 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.