Treatment strategies for rare diseases Glutaric acidemia type I, Maple syrup urine disease
8 years ago
We offer state-of-the-art preclinical magnetic resonance imaging and more than 10 years experience with animal models of rare metabolic disorders. We were the first to establish animal model of glutaric acidemia type I and to develop a treatment strategy. We have long-standing partnership with clinicians from USA specialized in management of rare metabolic disorders. In addition, as many metabolic disorders have a common theme, we have developed potential treatment for maple syrup urine disease. As we are an SME with no experience in regulatory affairs, we are looking for partners experienced in clinical trials that will develop the preclinical research results and bring treatment strategies from bench to bedside.
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