Larimar Given US Patent Protection for CTI-1601 Into 2040

Larimar Therapeutics has received a patent relative to CTI-1601, its experimental and potentially disease-modifying treatment for Friedreich’s ataxia (FA), the company announced. The patent, No. 11,459,363 and titled “Materials and Methods for Treating Friedreich’s Ataxia,” provides protection for composition of matter relative to CTI-1601 until at least July 2040.

G-CSF Stem Cell Therapy Safely Raises Frataxin Levels in Pilot Trial

Repeat use of G-CSF stem cell therapy, an approved medicine, was safe in adults with Friedreich’s ataxia and associated with significant elevations in frataxin protein and disease-related biomarkers, a pilot study in the U.K. reported. Because interventions that increase frataxin and reverse the harmful effects of the protein’s deficiency are…

A Conversation With Rare Disease Advocates