[visualization of where Dystrophin, the missing protein in DMD, belongs in the muscle fibers]

Project Catalyst is a first-of-its-kind research collaboration between Parent Project Muscular Dystrophy (PPMD) and PTC Therapeutics. The goal of Project Catalyst is to identify new treatments for all patients with DMD, regardless of the genetic mutation responsible for their disease. After critical support from families comprising PPMD’s Project Catalyst Circle and TCW Catalyst, the program was greatly accelerated by the participation of the National Institutes of Health (NINDS and NIAMS), which awarded a $15.4 million research grant in 2007. Since Project Catalyst was established in 2003, researchers have searched for compounds, or medicines, that address three key therapeutic targets, or proteins naturally produced by the body. Compounds that can adjust the level of these proteins may compensate for the loss of dystrophin and improve muscle growth, regeneration, and membrane stabilization. Preclinical testing of multiple compounds is currently underway in the mdx mouse, a commonly used animal model of DMD.

Target 1:
		Utrophin is similar to dystrophin, the protein that DMD patients are unable to produce in sufficient quantities. Researchers believe that utrophin could replace dystrophin if levels of the protein are sufficiently increased.
Target 2:
		Myostatin is a growth factor that regulates the size of muscles from early development through adulthood and prevents muscles from growing too large. Decreasing levels of myostatin in patients may stimulate muscle regeneration, increase muscle size, and improve strength.
Target 3:
		Insulin-like growth factor (IGF-1) plays an important role in the regeneration of damaged muscles by activating muscle cell proliferation and fusion. Increasing levels of IGF-1 in patients may help repair damaged muscles and preserve muscle function.

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