Neuromuscular Disease

Neuromuscular diseases are disabling, often fatal conditions characterized by the loss of muscle control, strength and function. Neuromuscular diseases affect both the voluntary muscles, which control the movement of our bodies (e.g. arms and legs), and autonomic muscles which subconsciously control activities like breathing. Unfortunately, there are limited treatment options for some of the most severe neuromuscular diseases. Acceleron is developing ACE-031, the lead candidate in its muscle program, to treat patients with:

Muscular Dystrophy
Amyotrophic Lateral Sclerosis (ALS)

Muscular Dystrophy

Muscular dystrophy (MD) refers to a group of genetic, hereditary muscle diseases that are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and deficiency of muscle cells and tissue.

The different MDs affect different muscle groups, have different ages of onset and cause different levels of disability and mortality. However, most types of MD are multi-system disorders that may affect skeletal muscles, the heart, gastrointestinal and nervous systems, eyes and other organs. Though estimates of its prevalence vary, there are probably more than 100,000 people in the United States affected by some form of MD.

ACE-031 is a new therapeutic in development that increases muscle mass and strength and has the potential to reverse the muscle loss in patients suffering from muscular dystrophy.

Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a progressive, fatal neuromuscular disease that destroys motor neurons, resulting in loss of muscle control and muscle strength. Patients ultimately lose the ability to start and control movement, leaving them paralyzed and eventually unable to breathe on their own.

There are approximately 25,000 ALS patients in the U.S.
Onset of ALS is generally from 40 to 70 years of age
Survival rate is low, and 75% of patients die within 5 years of diagnosis

ALS patients suffer from disability and poor quality of life

As ALS progresses, patients suffer from increasing difficulty with movement, swallowing, speaking, and using their hands and arms. Basic daily tasks, like standing or walking, and getting out of bed are taxing and eventually impossible. Atrophy of the diaphragm and respiratory muscles leads to respiratory failure, the primary cause of death in ALS patients.

Tremendous need for agents that can improve patient quality of life

Currently, only one therapy has been approved for the treatment of ALS. Though treatment may prolong survival and defer mechanical ventilation by several months, it does not improve patients’ physical function or quality of life, and it does not change the ultimate course of ALS.

Acceleron is developing ACE-031 , an agent that may be able to replenish muscle mass and strength lost to ALS. By building new muscle, ACE-031 may slow or actually reverse some of the devastating effects of ALS and improve physical function, slow the progression of disability, and ultimately improve patient quality of life.

Learn about ACE-031 for Muscle Growth and Repair