Saturday, August 10, 2024

Everything You Need to Know About Larimar Therapeutics' Nomlabofusp and the FDA's START Program

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August 7, 2024 By Andrew Cox, Pharm.D., MBA. www.managedhealthcareexecutive.com  Nomlabofusp is a protein replacement therapy designed to de...

Lexeo Therapeutics' LX2006 Shows Promise in Treating Friedreich Ataxia Cardiomyopathy

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August 9, 2024. By Andrew Cox, Pharm.D., MBA. Managed Healthcare Executive. Although the initial results are promising, LX2006's ultimat...

New and Emerging Drug and Gene Therapies for Friedreich Ataxia

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Scott V, Delatycki MB, Tai G, Corben LA. New and Emerging Drug and Gene Therapies for Friedreich Ataxia. CNS Drugs. 2024 Aug 8. doi: 10.1007...
Thursday, August 8, 2024

Glial cell activation precedes neurodegeneration in the cerebellar cortex of the YG8–800 murine model of Friedreich ataxia

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Andrés Vicente-Acosta, Saúl Herranz-Martín, María Ruth Pazos, Jorge Galán-Cruz, Mario Amores, Frida Loria, Javier Díaz-Nido, Glial cell acti...

Insulin sensitivity and insulin secretion in adults with Friedreich’s Ataxia: the role of skeletal muscle

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Jaclyn Tamaroff, Sara Nguyen, Neil E Wilson, Darko Stefanovski, Rui Xiao, Theresa Scattergood, Christopher Capiola, Gayatri Maria Schur, Jul...

Larimar Therapeutics Reports Second Quarter 2024 Operating and Financial Results

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BALA CYNWYD, Pa., Aug. 07, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) “We made significant achievements in our nomlabofus...

mTORC1 Signaling Inhibition Modulates Mitochondrial Function in Frataxin Deficiency

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mTORC1 Signaling Inhibition Modulates Mitochondrial Function in Frataxin Deficiency, Madison Lehmer, Roberto Zoncu, bioRxiv 2024.08.06.60694...
Friday, August 2, 2024

Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges

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Mishra, S., Venkatesh, M. Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges. Orphanet J Rare...
Wednesday, July 31, 2024

GLP-1R mediates idebenone-reduced blood glucose in mice

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Xin Zhao, Qingxuan Zeng, Siting Yu, Xiaochan Zhu, Bin Hu, Lijiao Deng, Yi Zhang, Yunfeng Liu,, GLP-1R mediates idebenone-reduced blood gluc...
Tuesday, July 30, 2024

Microfluidic formulation of diazoxide-loaded solid lipid nanoparticles as a Novel approach for Friedreich's ataxia treatment

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Ilaria Arduino, Antonella Santoro, Silvia De Santis, Rosa Maria Iacobazzi, Angela Assunta Lopedota, Eleonora Paradies, Giuseppe Merla, Sara ...