Wednesday, January 22, 2020

Expresión de IRS2 en ratones afectados con ataxia de Friedreich

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Gil Carceller, B. (2019). Grau en Biotecnologia, Universitat Politècnica de València. Escola Tècnica Superior d'Enginyeria Agronòmica i ...
Tuesday, January 21, 2020

Can this San Diego startup mimic exercise and fasting with a pill? Epirium raises $85M to find out

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San Diego Union Tribune, BRITTANY MEILING, JAN. 13, 2020 A biotech startup in La Jolla has raised $85 million from notable science invest...
Monday, January 20, 2020

Education and information needs for physicians about rare diseases in Spain

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Enrique Ramalle-Gómara, Elena Domínguez-Garrido, María Gómez-Eguílaz, María Eugenia Marzo-Sola, José Luis Ramón-Trapero & Josefa Gil-de-...
Sunday, January 19, 2020

Very-late-onset Friedreich’s ataxia: diagnosis in a kindred with late-onset cerebellar ataxia

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Conor Fearon, Roisin Lonergan, Damien Ferguson, Susan Byrne, David Bradley, Yvonne Langan, Janice Redmond; Neurology 2020;20:55-58. doi:10....
Saturday, January 18, 2020

Frataxin deficiency in Friedreich’s ataxia is associated with reduced levels of HAX-1, a regulator of cardiomyocyte death and survival

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Francesca Tiano, Francesca Amati, Fabio Cherubini, Elena Morini, Chiara Vancheri, Sara Maletta, Silvia Fortuni, Dario Serio, Andrea Quatrana...
Friday, January 17, 2020

PTC Therapeutics (PTCT) Presents At 38th Annual J.P. Morgan Healthcare Conference - Slideshow

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seekingalpha. Jan. 16, 2020 Slide deck was published by PTC Therapeutics, Inc. in conjunction with this event.
Thursday, January 16, 2020

Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public

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BioPharma Dive (WEB 01/16/2020) SAN FRANCISCO — Novartis sparked a new round of drug pricing criticism last May when the Swiss pharma reve...

Safety and Efficacy of Interferon γ in Friedreich's Ataxia

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Marinela Vavla MD PhD Maria Grazia D'Angelo MD PhD Filippo Arrigoni MD Nicola Toschi PhD Denis Peruzzo PhD Sandra Gandossini MD An...
Wednesday, January 15, 2020

Predictors of loss of ambulation in Friedreich's ataxia

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Christian Rummey, Jennifer M. Farmer, David R. Lynch; EClinicalMedicine, Volume 18, 2020, 100213, doi:10.1016/j.eclinm.2019.11.006. We pro...
Tuesday, January 14, 2020

PTC Therapeutics Provides Corporate Update and Highlights Pipeline Progress at 2020 J.P. Morgan Healthcare Conference

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SOUTH PLAINFIELD, N.J., Jan. 13, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today is providing a corporate update that will ...