Thelansis

Hutchinson-Gilford progeria syndrome is a genetic condition characterized by the dramatic, rapid appearance of aging beginning in childhood.  Affected children typically look normal at birth and in early infancy, but then grow more slowly than other children and do not gain weight at the expected rate (failure to thrive). Etiology- Mutations in the LMNA gene cause Hutchinson-Gilford progeria syndrome. The LMNA gene provides instructions for making a protein called lamin A. Epidemiology- The prevalence of HGPS is approximately 1 in 20 million, so at any given time, there are approximately 400 children living with progeria worldwide. As of December 2020, the Progeria Research Foundation International Progeria Registry has identified a total of 131 children and young adults living with progeria worldwide including 20 living in the US. The competitive landscape of Hutchinson-Gilford Progeria Syndrome includes country-specific approved and pipeline therapies. Any asset/product-specific ...