Rare Daily Staff The U.S. Food and Drug Administration gave Ultragenyx Pharmaceutical clearance to begin clinical trials of DTX401, an adeno-associated virus vector-based gene therapy for the treatment of glycogen storage disease type Ia, a genetic enzyme deficiency that result in the inability to regulate…Continue Reading
Search Results for: clinical trials
FDA Clears Ultragenyx to Begin Clinical Trials of Gene Therapy for Glycogen Storage Disease Type Ia
April 23, 2018 by
A Matchmaker for Patients Seeking Clinical Trials
March 15, 2018 by
In the past day, I’ve had several rare and common diseases, including some horrendous stuff, but all in the name of science. My sudden bout with a truckload of ailments came about when I learned that the American Liver Foundation had initiated an effort to…Continue Reading
Naia Rare Diseases Gets Green Light for Clinical Trials of Treatment for Short Bowel Syndrome
November 15, 2017 by
Rare Daily Staff Naia Rare Diseases, a biopharmaceutical company developing drugs for rare gastrointestinal diseases, said it has received authorization from the Medicine and Healthcare Products Regulatory Agency in the United Kingdom to initiate a clinical trial of NB 1001, its experimental therapeutic for adults…Continue Reading
FDA Awards 15 Grants for Clinical Trials to Stimulate Product Development for Rare Diseases
October 6, 2017 by
Rare Daily Staff The U.S. Food and Drug Administration said it awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to…Continue Reading
Rare Disease Report Interviews Janet Woodcock – Patients, Clinical Trials, and the FDA
October 26, 2016 by
At the NORD Rare Disease and Orphan Drug Breakthrough Summit in Arlington Virginia, we talked with Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA) about the increasing role patients have in the development…Continue Reading
Introducing 2016 Speaker Kent Thoelke: Leveraging Big Data to Grow Clinical Trials
September 15, 2016 by
As part of our 2016 Patient Advocacy Summit, we’re excited to have Kent Thoelke, Executive Vice President, Scientific & Medical Affairs, Safety & Commercialization at PRA Health Sciences as one of our dedicated speakers. He will also be attending our Tribute to Champions of Hope. We were were able to get…Continue Reading
RARECast: Using Social Media to Find Rare Disease Patients for Clinical Trials
July 22, 2016 by
Moving a rare disease therapy from the lab to the marketplace requires patients willing to participate in clinical trials that can demonstrate its safety and efficacy. But given the small number of people who may have a specific rare disease, finding these patients can slow…Continue Reading
.