LBSL, a rare genetic disorder that affects the brain and spinal cord, was first identified in 2004. While there are no treatments available today, researchers are working to understand the condition and develop therapies. One ongoing study at Kennedy Krieger Institute is using digital health…Continue Reading
Search Results for: clinical trials
Ferris Bueller and the Cost of Pivotal Clinical Trials
September 25, 2018 by
It turns out asking what a pivotal phase 3 clinical trial costs is a little like asking, “What does a car cost?” The answer will depend on whether the car in question is a $12,000 Nissan Versa, or a $13 million Rolls-Royce Sweptail. Like cars,…Continue Reading
FDA Awards 12 Grants to Fund Clinical Trials to Advance Rare Disease Treatments
September 25, 2018 by
Rare Daily Staff The U.S. Food and Drug Administration said that it has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to enhance the development of medical products for patients with rare diseases. “Developing a treatment…Continue Reading
FDA Clears Ultragenyx to Begin Clinical Trials of Gene Therapy for Glycogen Storage Disease Type Ia
April 23, 2018 by
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
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
.