Larimar Therapeutics, Inc. Updates on Nomlabofusp Development

On November 10, 2025, Larimar Therapeutics, Inc. provided updates on its nomlabofusp clinical program and future plans.

Individualized exercise and NAD+ precursor supplementation in Friedreich’s Ataxia: a randomized controlled trial

Lin, Kimberly Y. and Lin, Kimberly Y. and Bucha, Anna and Bucha, Anna and McSweeney, Kara and McSweeney, Kara and Wade, Kristin L. and Wade, Kristin L. and Karaj, Antoneta and Tamaroff, Jaclyn and Tamaroff, Jaclyn and O'Malley, Shannon and O'Malley, Shannon and Chung, Nicole M. and Chung, Nicole M. and Cilenti, Nicolette A. and Cilenti, Nicolette A. and Wanner, Julianne and Wanner, Julianne and Adzika, Gabriel K. and Adzika, Gabriel K. and Mesaros, Clementina and Blair, Ian A. and Blair, Ian A. and Rojsajjakul, Teerapat and Rojsajjakul, Teerapat and Serai, Suraj and Serai, Suraj and Farmer, Jennifer and Farmer, Jennifer and Bryant, Kyle and Bryant, Kyle and Lu, Yingying and Lu, Yingying and Harhay, Michael and Harhay, Michael and Weber, David R. and Weber, David R. and Paridon, Stephen M. and Paridon, Stephen M. and Seifert, Erin and Putt, Mary E. and Zamani, Payman and Zamani, Payman and Baur, Joseph A. and Lynch, David R. and Lynch, David R. and McCormack, Shana E. and McCormack, Shana E., Individualized exercise and NAD+ precursor supplementation in Friedreich’s Ataxia: a randomized controlled trial. doi:10.2139/ssrn.5698224. 

Interpretation: NR plus exercise for 12 weeks was safe and increased cardiovascular fitness in children and adults with FRDA. Adding NR to exercise could be considered as part of a comprehensive treatment approach.

The Triple Flexion Response in Friedrich’s Ataxia

Saluja A, Sahib A, Yadav V (November 04, 2025) The Triple Flexion Response in Friedrich’s Ataxia. Cureus 17(11): e96080. doi:10.7759/cureus.96080 

The triple flexion response is a significant diagnostic clue highlighting the marked corticospinal tract involvement in advanced Friedrich's ataxia.

Solid Biosciences Reports Third Quarter 2025 Financial Results

CHARLESTOWN, MA, Nov. 03, 2025 FA

(SGT-212): Solid has activated the first clinical trial site and is currently screening participants for FALCON, a Phase 1b first-in-human clinical trial evaluating SGT-212 for the treatment of Friedreich’s ataxia. 

SGT-212 for Friedreich’s Ataxia (FA)

In October 2025, the Company activated the first clinical trial site and began participant screening for FALCON, a first-in-human, open-label, Phase 1b clinical trial of SGT-212. The trial is expected to enroll non-ambulatory and ambulatory adult participants living with FA in up to three cohorts and is designed to evaluate the safety and tolerability of systemic and bilateral intradentate nucleus (IDN) administration of SGT-212. SGT-212 is the first investigational gene therapy for FA to utilize a dual route of administration and is intended to promote restoration of therapeutic levels of the frataxin protein to address the neurologic, cardiac and systemic clinical manifestations of FA.

Solid Biosciences Reports Third Quarter 2025 Financial Results

Early experience on omaveloxolone in adult patients with Friedreich's ataxia: a real-world observational study

Lima SM, Caltagirone M, Messina C, Quartetti U, Rini N, D'Amico F, Brighina F, Di Stefano V. Early experience on omaveloxolone in adult patients with Friedreich's ataxia: a real-world observational study. J Neurol. 2025 Nov 1;272(11):742. doi: 10.1007/s00415-025-13487-1. PMID: 41176519. 

 Omaveloxolone seems to be safe and well-tolerated in adult FRDA patients in the real-life setting. No significant worsening of symptoms was observed with no signs of progression, as well as the improvement of inflammatory biomarkers after 24 weeks of treatment, but no predictive factors for the disease response have been identified. However, the short duration, and the small sample size limit the generalizability of the results. Further studies with longer observation are needed to clearly define the efficacy of omaveloxolone in FRDA.

Friedreich ataxia:

Subramony SH, Lynch DR. Friedreich Ataxia. Pediatr Neurol. 2025 Nov 1;174:148-154. doi: 10.1016/j.pediatrneurol.2025.10.020. Epub ahead of print. PMID: 41252802.

With the introduction of potential new therapy for Friedreich ataxia (FRDA), the disorder has taken on a new importance in the world of pediatric neurology. Originally described more than 150 years ago, large scale clinical studies have defined diagnostic criteria and the underlying mutation as a biallelic, unstable expansion of an intronic GAA repeat in chromosome 9. In this review, we summarize the clinical features, routine management, pathophysiology, and emerging therapies for this devastating disease. The recent approval of omaveloxolone makes recognition of FRDA and its treatment essential for all pediatric neurologists.

Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix

Callenbach, M.H.E., van den Berg, S., Hulsbosch, A. et al. Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix. Orphanet J Rare Dis 20, 540 (2025). doi:10.1186/s13023-025-04020-8 

"New orphan medicinal products (OMPs) are increasingly expensive and approved with limited clinical evidence". 

The matrix provides a systematic approach applied to mitigate clinical and cost-effectiveness uncertainties and/or reimbursement challenges specific to OMPs through advising MEAs. This study highlights the diversity in the drivers of MEAs to reduce risk and reimbursement challenges specific to OMPs and underscores the relevance of considering both established and innovative MEAs to address these.

Positron emission tomography reveals increased myocardial glucose uptake in a subset of Friedreich ataxia patients

Payne, R.M., O’Connell, T.M., Pride, P.M. et al. Positron emission tomography reveals increased myocardial glucose uptake in a subset of Friedreich ataxia patients. Sci Rep 15, 37247 (2025). https://doi.org/10.1038/s41598-025-21330-w 

 In this cohort of FRDA patients, a PET scan identified two metabolically distinct subclasses of FRDA cardiomyopathy. FRDA patients with severe LVH had greater FDG uptake relative to palmitate utilization than FRDA patients without severe LVH. FRDA patients with severe LVH have systolic and diastolic dysfunction, as well as ongoing cardiac damage as indicated by cTnI leak. These findings suggest that FRDA patients with significant LVH may be at increased risk of complications from surgery or major illness.

Development of an AAV-Based Gene Therapy for the Ocular Phenotype of Friedreich’s Ataxia

Tang H, Gupte S, Xu E, Calabro KR, Friend H, Crosson SM, Fajardo D,Kostamo Z, Zhang H, Peterson JJ, Lin F, Kozmik Z, Lutz CM, Boye SL, Boye SE, Development of an AAV-Based Gene Therapy for the Ocular Phenotype of Friedreich’s Ataxia, Molecular Therapy (2025), doi:10.1016/j.ymthe.2025.10.048.

Gene supplementation via intravitreal injection of a novel AAV2-based capsid carrying FXN partially preserved retinal structure and/or function in both models, establishing proof-of-concept for this therapeutic strategy.

Neuropsychiatric challenges of Friedreich ataxia in a patient residing in a long-term care facility

Wong J, Kwok J, Kim K. Neuropsychiatric challenges of Friedreich ataxia in a patient residing in a long-term care facility. Prim Care Companion CNS Disord 2025;27(5):25cr03965. doi:10.4088/PCC.25cr03965 

 The neuropsychiatric manifestations of FA are often under-recognized despite their significant impact on patient care and quality of life. This case underscores the necessity for comprehensive management strategies that address both the neurological and behavioral aspects of FA. This patient residing in a long-term care facility exhibited chronic depression and significant behavioral challenges including refusal of food, medications, and nursing care, alongside physical aggression. Despite initial treatment with citalopram, behavioral symptoms persisted while the neurovegetative signs of depression improved. As the escalation of the patient’s behavioral symptoms marked a critical turning point, divalproex ER was introduced and titrated to manage his mood instability. Subsequently, the patient’s mood and behavior symptoms noticeably improved without worsening motor dysfunction, highlighting the importance of personalized psychopharmacologic interventions.