Valerion Therapeutics is utilizing a proprietary antibody-mediated delivery platform to target therapies to specific tissues via a well-known and broadly studied transport pathway.This platform is capable of enhanced intracellular delivery of a variety of active therapeutic payloads via a transport mechanism that is present in muscle and neurons and elevated in certain types of cancer. Pipeline candidates include therapeutic agents aimed at addressing a host of orphan genetic disorders with limited or no current therapies.
What is Pompe Disease?
Pompe disease is a rare multisystem genetic disorder that is characterized by absence or deficiency of the lysosomal enzyme alpha-glucosidase (GAA). This enzyme is required to breakdown (metabolize) the complex carbohydrate glycogen and convert it into the simple sugar glucose.delivery platform and pipeline>
What is Lafora Disease?
Lafora disease is an inherited, severe form of progressive myoclonus epilepsy. The condition most commonly begins with epileptic seizures in late childhood or adolescence. Other signs and symptoms include difficulty walking, muscle spasms (myoclonus) and dementia.delivery platform and pipeline>
What is Myotubular Myopathy?
X-linked myotubular myopathy is a condition that primarily affects muscles used for movement (skeletal muscles) and occurs almost exclusively in males. People with this condition have muscle weakness (myopathy) and decreased muscle tone (hypotonia) that are usually evident at birth.delivery platform and pipeline>
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