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....
Wednesday, April 14, 2021

Remember friedreich ataxia even in a toddler with apparently isolated dilated (not hypertrophic!) cardiomyopathy: revisited

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Anwar BABAN, Marianna CICENIA, Lorena TRAVAGLINI, Federica CALÍ, Gessica VASCO, Paola FRANCALANCI, Antonio NOVELLI, Rachele ADORISIO, Antoni...
Tuesday, April 13, 2021

The Cardioprotective Mechanism of Phenylaminoethyl Selenides (PAESe) Against Doxorubicin-Induced Cardiotoxicity Involves Frataxin

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Fu Xiaoyu, Eggert Mathew, Yoo Sieun, Patel Nikhil, Zhong Juming, Steinke Ian, Govindarajulu Manoj, Turumtay Emine Akyuz, Mouli Shravanthi, P...
Monday, April 12, 2021

Diseases Costs and Impact of the Caring Role on Informal Carers of Children with Neuromuscular Disease

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Rodríguez, A.A.; Martínez, Ó.; Amayra, I.; López-Paz, J.F.; Al-Rashaida, M.; Lázaro, E.; Caballero, P.; Pérez, M.; Berrocoso, S.; García, M....
Friday, April 9, 2021

A Phase 2a investigator-initiated open-label clinical trial assessing elamipretide

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BOSTON, April 6, 2021 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the dis...