Saturday, March 7, 2026

Health care, social, educational, and employment needs of individuals affected by rare diseases and their families in Catalonia, Spain

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Calmaestra-Carrillo, E., Vernet, R., Torrent-Farnell, J. et al. Health care, social, educational, and employment needs of individuals affect...

FDA reversals leave investors worrying about the fates of other experimental drugs

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Published Fri, Mar 6 2026. The FDA in the past year has denied or discouraged applications of at least eight new drugs, according to RTW Inv...
Thursday, February 26, 2026

Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026

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BALA CYNWYD, Pa., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology comp...
Wednesday, February 18, 2026

Solid Biosciences Highlights FDA Path for SGT-003 DMD Study, Shares Early Friedreich’s Ataxia Update

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Defense World Staff on Feb 17th, 2026 Friedreich’s ataxia: dual route administration and first patient dosed Turning to FA, Cumbo said the p...
Saturday, February 14, 2026

Targeting frataxin deficiency in DRG neurons and fibroblasts: omaveloxolone restores metabolic and iron balance to reduce ferroptosis

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Portillo-Carrasquer M, Sanz-Alcázar A, Sánchez-López B, Delaspre F, Pazos-Gil M, Oliveira-Jorge L, Castells-Roca L, Tamarit J, Ros J, Cabisc...
Wednesday, February 11, 2026

Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale

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Rummey C, Farmer JM, Lynch DR. Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale. Ann Clin Transl Neurol...
Sunday, February 8, 2026

Syndromic and Metabolic Cardiomyopathies: Rare Variants for Adults (Glycogen Storage Diseases, Friedreich’s Ataxia)

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Turk Kardiyol Dern Ars 2024;52(Suppl 1):S1-S206. In Friedreich’s Ataxia patients, concentric left ventricular hypertrophy is common; asymmet...
Tuesday, February 3, 2026

Clinical manifestations of Friedreich ataxia in patient with neurofibromatosis seen at Daoud Charity Clinic Sudan: A case report

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Abbasher Hussein, Mohajer Ismael, Mohamed Baraka, Abdaleliah Abobker, Wadah Altoom, Osama Suliman, Clinical manifestations of Friedreich ata...

Mitochondrial iron overload is associated with lysosomal dysfunction-mediated mitophagy impairment in the heart of Friedreich’s ataxia

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Eunbin Jee, Maisha Medha, Hwayoung Baek, Jonghan Kim, Yuho Kim, Mitochondrial iron overload is associated with lysosomal dysfunction-mediate...
Monday, February 2, 2026

La omaveloxolona recibe luz verde en España como primer tratamiento específico para la Ataxia de Friedreich

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Comisión Interministerial de Precios de los Medicamentos 28 de enero de 2026. Skyclarys H* (omaveloxolona): Tratamiento de la ataxia de Frie...