We’re putting muscle into finding newtreatments
We are dedicated to the research and development of first-in-class therapies for people suffering from debilitating diseases that rob them of key muscle functions. We are passionate about advancing muscle biology-driven treatments for diseases that are characterized by compromised muscle function, muscle weakness and fatigue, like heart failure, amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). By focusing on improving the mechanics of muscle with investigational medicines that may increase strength, power, and performance, we aspire to develop new treatment options that may dramatically improve the lives and functionality of people living with debilitating diseases.
Muscles power the essential aspects of our lives, from breathing to movement. When muscles are weakened by disease, even normal daily activities can be quite challenging and can severely limit a person’s independence.
In ALS, the progressive death of motor neurons results in diminished stimulation of muscles that perform vital bodily functions, beginning with simple movement and eventually resulting in the loss of respiratory function.
The rates of morbidity and mortality following a diagnosis of heart failure are unacceptably high despite current treatments that do not adequately address impaired cardiac muscle function.
SMA is a serious genetic disease characterized by severe and progressive muscle weakness which affects large and fine motor skills, limiting mobility and ultimately breathing.