Company Overview - Larimar Therapeutics is focused on developing treatments for Friedreich's ataxia, a rare neurodegenerative disease affecting approximately 5,000 patients in the United States and around 20,000 globally [4][5] - The disease primarily affects pediatric patients, with about 70% of diagnoses occurring before the age of 14, leading to progressive deterioration and early mortality [4] Disease Mechanism - Friedreich's ataxia is caused by a deficiency in frataxin, a protein crucial for mitochondrial function [5] - Patients typically have only 20% to 40% of normal frataxin levels, with severely affected individuals having as low as 5% to 10% [5] - Heterozygous carriers, such as the parents of affected children, do not exhibit symptoms of the disease [5]
Larimar Therapeutics, Inc. (LRMR) Presents at 44th Annual J.P. Morgan Healthcare Conference Transcript