Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Saint Louis University’s (SLU) Center for World Health and Medicine, announced that they… Read More »
Ultragenyx Pharmaceutical to sponsor FSHD drug development project
Whole-Body MRI Yields New Insights
By June Kinoshita (from FSH Watch Fall 2015 issue) Click on image below to view full size. In 2013-2014, the FSH Society funded a study led by Doris Leung, MD,… Read More »
Acceleron plans to launch Phase 2 FSHD trial this year
According to a Boston Business Journal story, Cambridge, Massachusetts-based biotech Acceleron Pharma has plans to test its ACE-083 compound in facioscapulohumeral muscular dystrophy patients in a phase 2 clinical trial later… Read More »
Update on Early-Onset FSH Muscular Dystrophy Study
Insights on symptoms, biology, and quality of life By Yi-Wen Chen, PhD and Jean Mah, MD While most people with FSHD develop muscle weakness during late teen years or adulthood,… Read More »
Biology 101 for understanding the CRISPR research
Peter Jones, PhD, and Charis Himeda, PhD, found themselves unexpectedly in the media spotlight last week after publication of their paper on the application of CRISPR technology in FSHD cells. In… Read More »