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Larimar Nears Data Readout For Nomlabofusp In Rare Inherited Disorder

By RTTNews Staff Writer   ✉  | Published:  | Google News Follow Us  | Join Us
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Larimar Therapeutics Inc. (LRMR), which is developing a drug for a rare inherited condition, has clinical data readouts expected later this year that investors should watch closely.

The company's lead drug candidate is Nomlabofusp, being developed as a potential disease-modifying therapy for Friedreich's ataxia.

Friedreich's ataxia (FA) is a rare inherited condition caused by a mutation in FXN, a gene that provides instructions for making the mitochondrial protein frataxin. Most individuals with FA produce only about 20-40% of normal frataxin (FXN) levels. This genetic disorder causes progressive damage to the nervous system, resulting in motor weakness and sensory loss. About 20,000 patients worldwide are affected by FA, including around 5,000 in the United States. The majority of the remaining cases are found in Europe. Notably, nearly 70% of patients are diagnosed before the age of 14.

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