PTC Therapeutics Inc. has formed an orphan disease research collaboration with the Perelman School of Medicine at the University of Pennsylvania.
The academic-industry partnership will focus on discovering and developing new treatments for rare diseases.
Stuart Peltz, CEO of PTC Therapeutics in South Planfield, N.J., said their first project will be to look at Translarna as a potential treatment for Mucopolysaccharidosis-I, an inherited lysosomal storage disorder caused by a deficiency of an enzyme involved in the breakdown of complex carbohydrates.
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