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Genetic Disorder Services In Usa Virginia

1 services found
Developing Treatments for Rare Diseases

Developing Treatments for Rare Diseases

Manufactured by:HemoShear Therapeutics, Inc.   based inCharlottesville, VIRGINIA (USA)
In our most advanced program, the company is conducting a phase 2 clinical trial of HST5040, an oral small molecule investigational drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). These genetic disorders are caused by deficiencies of certain metabolic enzymes that lead to buildup of life-threatening toxins. ...
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