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Acceleron Pharma's ACE-031 Increases Muscle Mass and Strength in,Preclinical Studies

skeletal muscle will offer a growing number of patients an excellent opportunity to benefit from a promising therapy for the otherwise untreatable muscle loss associated with their disease," said Matthew L. Sherman, M.D., Chief Medical Officer of Acceleron. "Based on the comprehensive preclinical program currently underway, Acceleron is working toward advancing ACE-031 into Phase 1 clinical trials later this year."

Study Findings

The presentation, entitled "Treatment with a Soluble Activin Receptor IIB Causes Increased Muscle Mass and Strength in mdx Mice" discussed data from preclinical studies in normal mice and in a mouse model of muscular dystrophy. These studies show a substantial increase in muscle mass and a dose-dependent increase in lean body mass in treated animals. These effects are significant in just two weeks following start of therapy and increase further as treatment continues. In the mdx murine model of muscular dystrophy, treated animals showed increases in muscle mass which translated into significantly increased muscle strength.

About Muscle Loss

Loss of muscle mass and strength is a serious clinical consequence of several types of diseases and conditions: (1) neuromuscular diseases, such as ALS and muscular dystrophy, in which patients suffer from the loss of muscle strength and function which ultimately leads to respiratory failure and death (2) cancer-cachexia, in which patients can lose muscle mass and body weight as a result of both their cancer treatments (hormone deprivation therapy causes loss of muscle) and the cancer itself (3) other cachexias, in which the muscle loss is associated with chronic diseases such as congestive heart failure, chronic obstructive pulmonary disease, and chronic kidney disease and, (4) age-related muscle loss, known as sarcopenia, in which generalized muscle loss and frailty leads to frequent falls and morbidity and mortality from broken bones. A drug candidate that would increase m
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