Pompe Disease is a genetic disorder that is caused by building up of glycogen in body’s organs and tissues. This further leads to impairment of the ability to function normally. According to the researchers, there are three types of pompe disease, including non-classic infantile-onset, late-onset, and classic infantile-onset. The non-classic infantile-onset pompe disease is generally characterized by delayed motor skills. The classic form of infantile-onset pompe disease causes myopathy, hypotonia, and hepatomegaly. The late-onset type of pompe disease is generally milder than infantile-onset forms and causes progressive muscle weakness. Valerion Therapeutics, LLC is in the process of developing VAL-1221 for the treatment of pompe disease. Sanofi SA is in the process of developing GZ402666 for the treatment of pompe disease. Some of the companies and universities having the pipeline of pompe disease include University of Florida, Sanofi SA, and others.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.