Acceleron Receives FDA Orphan Drug Designation for ACE-083

From BusinessWire CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation for ACE-083, the Company’s locally acting “Myostatin+” muscle agent, for the treatment of … Read more of “Acceleron Receives FDA Orphan Drug Designation for ACE-083” »

2018 FSHD Connect videos!

Our 2018 FSHD Connect conference was held at the Flamingo Las Vegas on June 9-10, 2018. The conference gathered together 400 patients, family members, researchers, physicians, and health experts for a day-and-a-half of intensive learning and community activism. Our meeting featured talks by thought leaders on the latest in scientific research and medical management of … Read more of “2018 FSHD Connect videos!” »

FDA orphan drug designation for Genea Biocells’ FSHD drug candidate

Genea Biocells, a San Diego-based biotech company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Scientists from Genea Biocells will be attending the FSH … Read more of “FDA orphan drug designation for Genea Biocells’ FSHD drug candidate” »

A new tool for measuring disease burden in FSH muscular dystrophy

 by Amanda Hill, Denver, Colorado FSHD clinical researchers recently published what may soon become a standard battery of assessments for use in clinical trials, an exciting and essential milestone for the development of FSHD therapies. The combined assessments are altogether called a “composite outcome measure” (COM) and are designed to more fully capture changes in … Read more of “A new tool for measuring disease burden in FSH muscular dystrophy” »

Acceleron Receives FDA Fast Track Designation for its FSHD drug

The Massachusetts-based biotech, Acceleron Pharma, issued a press release this morning with some encouraging news for FSH muscular dystrophy patients about its experimental drug, ACE-083 (see related story). Here is what it says: CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and … Read more of “Acceleron Receives FDA Fast Track Designation for its FSHD drug” »

ACE-083 Phase 2 trial results presented at AAN conference

Preliminary results from the ongoing phase 2 clinical trial of ACE-083 in FSHD patients were presented today at the American Academy of Neurology 70th Annual Meeting in Los Angeles, California.The oral presentation by Jeffery Statland, MD, of the University of Kansas Medical Center, included preliminary findings from cohorts 1 and 2 in the open-label, dose … Read more of “ACE-083 Phase 2 trial results presented at AAN conference” »