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Akashi is currently treating patients with DMD in a phase 1b/2a multi-center clinical program to evaluate HT-100 safety and tolerability and assess trends in a range of exploratory biomarkers and efficacy endpoints. About HT-100 HT-100 (delayed-release halofuginone) is an orally available, small molecule drug candidate designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation for DMD in both the U.S. and EU, and fast track designation in the U.S. About Duchenne muscular dystrophy (DMD) Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties. About Akashi Therapeutics Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.
Keywords for this news article include: Neurology, FDA Actions, Inflammation, Muscular Dystrophies, Neuromuscular Diseases, Inborn Genetic Diseases, Nervous System Diseases,
Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC
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