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” »

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” »

Acceleron Announces Preliminary Results from Part 1 of the ACE-083 Phase 2 Trial in Patients with Facioscapulohumeral Dystrophy

We are sharing the following press release from Acceleron Pharma, Inc., regarding the progress of their Phase 2 clinical trial of ACE-083. The FSH Society collaborates with Acceleron to help the company understand patients’ symptoms, motivations, desired outcomes, etc.  – Mean total muscle volume increases of over 12% in the tibialis anterior and biceps brachii … Read more of “Acceleron Announces Preliminary Results from Part 1 of the ACE-083 Phase 2 Trial in Patients with Facioscapulohumeral Dystrophy” »

Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes

Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain Critical Diagnostic Classification Standard HACKENSACK, N.J., Dec. 7, 2017 /PRNewswire-USNewswire/ — Parent Project Muscular Dystrophy (PPMD), along with collaborators the FSH Society and the Foundation to Eradicate Duchenne (FED), are pleased to announce that the nominations to create more specific ICD-10 codes for Duchenne/Becker muscular … Read more of “Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes” »

aTyr Pharma releases results of early-onset FSHD trial

SAN DIEGO, April 24, 2017 (GLOBE NEWSWIRE) — aTyr Pharma, Inc. (Nasdaq:LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced promising clinical results from its Phase 1b/2 003 trial assessing the safety and potential activity of Resolaris™ in patients with early onset facioscapulohumeral muscular … Read more of “aTyr Pharma releases results of early-onset FSHD trial” »

Acceleron’s ACE-083 clinical trial is now open

Cambridge, Massachusetts-based Acceleron Pharma, Inc., is now recruiting adults with FSH muscular dystrophy for a Phase 2 clinical trial of ACE-083. The initial trial site is located in St. Louis, Missouri. Additional sites across the U.S. are expected to begin recruiting soon. ACE‐083 is an investigational drug that inhibits selected proteins in the transforming growth … Read more of “Acceleron’s ACE-083 clinical trial is now open” »

aTyr Pharma Receives FDA Fast Track Designation for Resolaris™

— First Reported Fast Track Designation for a FSHD Treatment — SAN DIEGO, Oct. 24, 2016 /PRNewswire/ — aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced that its product candidate Resolaris™ was granted Fast Track designation by the US Food … Read more of “aTyr Pharma Receives FDA Fast Track Designation for Resolaris™” »