本文是关于 frataxin 蛋白的综述，该蛋白与弗里德赖希共济失调（Friedreich’s ataxia）这一罕见病相关。文章聚焦其折叠、结构、动力学和功能等生物物理与生物化学特性，探讨其在疾病中的作用、治疗方法，还研究了它的系统发育分布。 一、引言 frataxin 是一种与 ...

Lexeo Therapeutics has posted updated data in Friedreich ataxia (FA) patients, linking a gene therapy to improvements on ...

Lexeo reported strong interim results for LX2006 in ataxia cardiomyopathy, with plans to start a registrational study by early 2026.

The firm plans to use improvement in left ventricular mass index and increased frataxin protein expression after LX2006 treatment as co-primary endpoints.

Investing.com — 周二，花旗重申对Larimar Therapeutics (NASDAQ: LRMR )股票的买入评级，目标价为14.00美元。根据 InvestingPro 数据，这一目标价较当前2.28美元的价格有显著上涨空间，该股目前交易价接近其52周低点2.25美元，而52周高点为11.20美元。花旗最新评论紧随Larimar宣布美国食品药品监督管理局 ...

Lexeo Therapeutics (LXEO) announced positive interim data across all dose cohorts of LX2006 for the treatment of Friedreich ataxia ...

Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced significant progress in its nomlabofusp program for Friedreich’s ...

frataxin expression to be evaluated for any increase from baseline rather than numerical threshold Interim update from cohort 1 of LX2020 HEROIC-PKP2 Phase 1/2 trial: observed 71% and 115% ...

Additional alignment with FDA on LX2006 planned pivotal study including protein expression co-primary endpoint: based on improvements in LVMI across participants with abnormal LVMI at baseline, ...