larimartx.comCrawled Oct 25, 2026
Larimar Therapeutics is a clinical-stage biotechnology company focused on treating rare diseases, with its lead candidate, nomlabofusp, in a Phase 2 trial for Friedreich’s ataxia.
Nomlabofusp, discovered by R. Mark Payne, MD, is a recombinant fusion protein designed to deliver human frataxin into the mitochondria of Friedreich’s ataxia patients, who cannot produce enough of this essential protein due to a genetic abnormality.
Molecular Genetics and Metabolism | Friedreich ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by reduced amounts of the mitochondrial protein frataxin. Frataxin levels in research studies are typically measured via Western blot analysis from patient fibroblasts, lymphocytes, or muscle biopsies; none of these is ideal for rapid detection in large scale clinical studies.
Friedreich’s ataxia (FRDA) is the most common inherited human ataxia, caused by a deficiency of the mitochondrial protein frataxin (FXN), which leads to progressive ataxia and cardiomyopathy.
Riley Symposium | Friedreich’s ataxia is a multisystem disorder of mitochondrial function affecting primarily the heart and brain. Patients experience a severe cardiomyopathy that can progress to heart failure and death.
Larimar did not sponsor the publications listed below and is including them for informational purposes only.
BALA CYNWYD, Pa., Sept. 29, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the appointment of John Harlow as President and Chief Operating Officer,
Larimar Therapeutics is a company based in the Philadelphia area that develops treatments to improve the lives of patients with rare diseases.
Larimar Therapeutics develops treatments for complex rare diseases, initially focusing on Friedreich’s ataxia. Its protein replacement therapy platform aims to deliver missing proteins inside cells to address diseases with limited or no existing treatments.
Friedreich’s Ataxia Research Alliance (FARA) is a nonprofit organization dedicated to funding research, raising awareness, and collaborating with scientists, patients, clinicians, and pharmaceutical companies to develop treatments and a cure for Friedreich’s ataxia.
Three Bala Plaza East Suite 506 Bala Cynwyd, PA 19004 844-511-9056
Three Bala Plaza East Suite 506 Bala Cynwyd, PA 19004
Clinical-stage biotechnology company focused on developing treatments for complex rare diseases.