Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]
Neuroinflammation is inflammation of the nervous tissue. It may be initiated in response to a variety of cues, including infection, traumatic brain injury, toxic metabolites, or autoimmunity. In the CNS, including the brain and spinal cord, microglia are the resident innate immune cells that are activated in response to these cues. The CNS is typically an immunologically privileged site because peripheral […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases Due to biological’s unique characteristics, study designers may find that results in pre-clinical trials do not predict acute, chronic and late onset immunogenicity and safety. In Addition, the biological orphan has a long PK half-life of months such as with any mAb, […]
