Saturday, September 5, 2020

Broad Institute launches new effort to study rare neuromuscular disorder

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Broad Institute, NEWS / 09.3.20. By Leah Eisenstadt. A new research and drug discovery effort at the Broad Institute of MIT and Harvard is ...
Friday, September 4, 2020

Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019

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Xavier Badia, Tania Vico, John Shepherd, Alicia Gil, José Luis Poveda-Andrés & César Hernández; Orphanet J Rare Dis 15, 224 (2020). doi:...
Thursday, September 3, 2020

AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy

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Novartis, Sep 02, 2020 Novartis has renamed the previously acquired AveXis to Novartis Gene Therapies. The change signifies the growing im...

Reata Announces the Presentation of the Pivotal MOXIe Part 2 Study of Omaveloxolone in Friedreich’s Ataxia at the American Academy of Neurology

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September 03, 2020 06:45 ET | Source: Reata Pharmaceuticals, Inc. PLANO, Texas, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, ...

High levels of frataxin overexpression leads to mitochondrial and cardiac toxicity in mouse models

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Brahim Belbellaa, Laurence Reutenauer, Nadia Messaddeq, Laurent Monassier, Hélène Puccio; Molecular Therapy - Methods & Clinical Develop...
Wednesday, September 2, 2020

The uses of resveratrol for neurological diseases treatment and insights for nanotechnology based-drug delivery systems

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Bruno Fonseca-Santos, Marlus Chorilli, International Journal of Pharmaceutics, 2020, 119832, doi:10.1016/j.ijpharm.2020.119832. Due to the...
Tuesday, September 1, 2020

An Optimally Weighted User- and Item-based Collaborative Filtering Approach to Predicting Baseline Data for Friedreich’s Ataxia Patients

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Wenbin Yue, Zidong Wang, Weibo Liu, Bo Tian, Stanislao Lauria, Xiaohui Liu, Neurocomputing, 2020, doi:10.1016/j.neucom.2020.08.031. In thi...
Monday, August 31, 2020

Plasma and red blood cell membrane accretion and pharmacokinetics of RT001 (bis-allylic 11,11-D2-linoleic acid ethyl ester) during long term dosing in patients

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J. Thomas Brenna, Genevieve James, Mark Midei, Frederic Heerinckx, Paldeep Atwal, Peter Milner, Karsten Schmidt, Lex van der Ploeg, Robert F...
Monday, August 24, 2020

Friedreich Ataxia: current state-of-the-art, and future prospects for mitochondrial-focused therapies

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Federico V. Pallardó, Giovanni Pagano, Laura R. Rodríguez, Pilar Gonzalez-Cabo, Alex Lyakhovich, Marco Trifuoggi; Translational Research, 2...
Sunday, August 23, 2020

Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

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Pietro Giuseppe Mazzara, Sharon Muggeo, Mirko Luoni, Luca Massimino, Mattia Zaghi, Parisa Tajalli-Tehrani Valverde, Simone Brusco, Matteo Ja...