Thelansis

 Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, progressively debilitating, and fatal disease caused by pathogenic mutations in the transthyretin (TTR) gene that result in the accumulation of amyloid fibrils throughout the body, including in the heart, peripheral nerves, and gastrointestinal tract. This accumulation leads to damage to organs and tissue, including peripheral sensorimotor neuropathy, autonomic neuropathy, and cardiomyopathy, leading to decreased quality of life and eventually death. Most patients with hATTR amyloidosis develop a mixed phenotype of both polyneuropathy and cardiomyopathy due to this multisystem involvement. ·        hATTR amyloidosis affects approximately 56,000 to 74,500 people worldwide and has a median survival of 5 to 7 years following diagnosis, with reduced survival of 3 to 4 years for patients presenting with cardiomyopathy.   Thelansis’s “Hereditary ATTR Amyloidosis Market Outlook, Epid...

 Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, progressively debilitating, and fatal disease caused by pathogenic mutations in the transthyretin (TTR) gene that result in the accumulation of amyloid fibrils throughout the body, including in the heart, peripheral nerves, and gastrointestinal tract. This accumulation leads to damage to organs and tissue, including peripheral sensorimotor neuropathy, autonomic neuropathy, and cardiomyopathy, leading to decreased quality of life and eventually death. Most patients with hATTR amyloidosis develop a mixed phenotype of both polyneuropathy and cardiomyopathy due to this multisystem involvement. ·        hATTR amyloidosis affects approximately 56,000 to 74,500 people worldwide and has a median survival of 5 to 7 years following diagnosis, with reduced survival of 3 to 4 years for patients presenting with cardiomyopathy. Thelansis’s “Hereditary ATTR Amyloidosis Market Outlook, Epidemiology,...