Monday, May 10, 2021

Larimar Therapeutics Reports First Quarter 2021 Operating and Financial Results

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BALA CYNWYD, Pa., May 10, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology com...
Friday, May 7, 2021

Reata Pharmaceuticals, Inc. Announces First Quarter 2021 Financial Results and Provides an Update on Clinical Development Programs

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May 06, 2021, Source: Reata Pharmaceuticals, Inc.  Data from the registrational Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone i...

Drp1-dependent peptide reverse mitochondrial fragmentation, a homeostatic response in Friedreich ataxia

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Johnson J, Mercado-Ayón E, Clark E, Lynch D, Lin H.; Pharmacol Res Perspect. 2021 May;9(3):e00755. doi: 10.1002/prp2.755. PMID: 33951329. T...
Thursday, May 6, 2021

Scoliosis in Friedreich's ataxia: longitudinal characterization in a large heterogeneous cohort

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Rummey C, Flynn JM, Corben LA, Delatycki MB, Wilmot G, Subramony SH, Bushara K, Duquette A, Gomez CM, Hoyle JC, Roxburgh R, Seeberger L, Yoo...
Monday, May 3, 2021

A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)

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Fernando de Andrés-Nogales, Encarnación Cruz, Miguel Ángel Calleja, Olga Delgado, Maria Queralt Gorgas, Jaime Espín, Jorge Mestre-Ferrándiz,...
Sunday, May 2, 2021

Revisión Bibliográfica: Ataxia de Friedreich y sus manifestaciones cardiovasculares

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Santiago Andrés Vintimilla Pesántez; Ocronos. Vol. IV. Nº 4–Abril 2021. Pág. Inicial: Vol. IV; nº4: 150. ORCID: https://orcid.org/ 0000-0003...
Thursday, April 29, 2021

LEXEO Therapeutics Announces Upcoming Data Presentations at the American Society of Gene and Cell Therapy (ASGCT) 2021 Virtual Annual Meeting

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NEW YORK, April 28, 2021 (GLOBE NEWSWIRE) -- LEXEO Therapeutics, a clinical-stage gene therapy company, today announced upcoming presentatio...
Wednesday, April 28, 2021

Frataxin deficiency promotes endothelial senescence in pulmonary hypertension

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Miranda K. Culley, Jingsi Zhao, Yi Yin Tai, Ying Tang, Dror Perk, Vinny Negi, Qiujun Yu, Chen-Shan C. Woodcock, Adam Handen, Gil Speyer, Seu...
Tuesday, April 27, 2021

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

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CAMBRIDGE, MASS. AND SOMERSET, N.J. (PRWEB) APRIL 27, 2021  AavantiBio, a gene therapy company focused on transforming the lives of patients...
Sunday, April 18, 2021

Longitudinal structural brain changes in Friedreich ataxia depend on disease severity: the IMAGE-FRDA study

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Selvadurai LP, Georgiou-Karistianis N, Shishegar R, Sheridan C, Egan GF, Delatycki MB, Harding IH, Corben LA.; J Neurol (2021). https://doi....