Monday, February 12, 2024

Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia

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CAMBRIDGE, Mass., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized SKYCLA...
Friday, February 9, 2024

Real-world evidence for coverage determination of treatments for rare diseases

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Dayer, V.W., Drummond, M.F., Dabbous, O. et al. Real-world evidence for coverage determination of treatments for rare diseases. Orphanet J R...
Tuesday, February 6, 2024

Searching for Frataxin Function: Exploring the Analogy with Nqo15, the Frataxin-like Protein of Respiratory Complex I from Thermus thermophilus

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Doni, D.; Cavallari, E.; Noguera, M.E.; Gentili, H.G.; Cavion, F.; Parisi, G.; Fornasari, M.S.; Sartori, G.; Santos, J.; Bellanda, M.; et al...

Safety, pharmacokinetics, and pharmacodynamics of nomlabofusp (CTI-1601) in Friedreich's ataxia

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Clayton, R., Galas, T., Scherer, N., Farmer, J., Ruiz, N., Hamdani, M., Schecter, D. and Bettoun, D. (2024), Safety, pharmacokinetics, and p...
Monday, February 5, 2024

Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: a clinically silent peripheral neuropathy

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Cheryl Tyszkiewicz, Seo-Kyoung Hwang, Jamie K. DaSilva, Ramesh C. Kovi, Kelly A. Fader, Madhu P. Sirivelu, June Liu, Chris Somps, Jon Cook, ...
Sunday, February 4, 2024

A peptide derived from TID1S rescues frataxin deficiency and mitochondrial defects in FRDA cellular models

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Yi Na Dong, Lucie V. Ngaba, Jacob An, Miniat W. Adeshina, Nathan Warren, Jonathan Wong, David R. Lync, Front. Pharmacol. Sec. Neuropharmacol...

Nerve Ultrasound in Friedreich’s Ataxia: enlarged nerves as a Biomarker of disease severity

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G. Di Pietro, E. Cioffi, P. Falco, E. Galosi, G. De Stefano, G. Di Stefano, C. Leone, V. Martines, S. Perotti, C. Casali, A. Truini, Nerve U...
Tuesday, January 30, 2024

Protective effect of FXN overexpression on ferroptosis in L-Glu-induced SH-SY5Y cells

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Mengran Wang, Tingting Xuan, Haining Li, Jing An, Tianhui Hao, Jiang Cheng, Protective effect of FXN overexpression on ferroptosis in L-Glu-...
Friday, January 26, 2024

Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression

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Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression, Negin Mozafari, Salome Isabel...

Omaveloxolone for the treatment of Friedreich Ataxia: clinical trial results and practical considerations

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David R Lynch, Susan Perlman & Kim Schadt (2024) Omaveloxolone for the treatment of Friedreich Ataxia: clinical trial results and practi...