Luke Rosen is the father of a daughter with KIF1A associated neurological disorder (KAND) and co-founder of the highly successful patient organization, KIF1A.org. Rosen also works with Ovid Therapeutics as their Vice President of Patient Engagement & Government Affairs, so he understands what patient groups want from industry. In this video, Rosen provides some insight on how patient groups can best proceed in partnering with the pharmaceutical industry.
KAND is due to a mutation in the KIF1A gene. Currently, the disease is not well understood, partly due to the fact that there is a large variance in symptoms that occur in these children. Some of the more common symptoms include spastic paraplegia, hypotonia, ataxia, intellectual disability, epilepsy, optic nerve atrophy, cerebellar atrophy, peripheral neuropathy, and autistic-like behaviour.
Ovid Therapeutics is a biotech company focused on developing treatments for a number of rare conditions, including angelman syndrome, fragile X syndrome, and dravet syndrome.