Saturday, September 12, 2020

Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich’s Ataxia Patients

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Pérez-Luz, S.; Loria, F.; Katsu-Jiménez, Y.; Oberdoerfer, D.; Yang, O.-L.; Lim, F.; Muñoz-Blanco, J.L.; Díaz-Nido, J.; Int. J. Mol. Sci. 202...
Friday, September 11, 2020

Iron-Sulfur Cluster Complex Assembly in the Mitochondria of Arabidopsis thaliana

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  Alejandro M. Armas, Manuel Balparda, Agustina Terenzi, Maria V. Busi, Maria A. Pagani and Diego F. Gomez-Casati; Plants 2020, 9(9), 1171, ...
Thursday, September 10, 2020

Rare Disease Trials Require Interactions With KOLs, Patients, & Regulators

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  Clinical Leader, September 9, 2020; Chief Editor: Ed Miseta Minoryx Therapeutics is a small biotech hoping to bring new hope to patients...
Tuesday, September 8, 2020

Ataxia: Hope starts with measurement

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The Florey Institute of Neuroscience and Mental Health. News and media. 03 Sep 2020. The team will be developing a prototype device to meas...

Molecular and Cellular Substrates for the Friedreich Ataxia. Significance of Contactin Expression and of Antioxidant Administration

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Bizzoca, A.; Caracciolo, M.; Corsi, P.; Magrone, T.; Jirillo, E.; Gennarini, G.; Molecules 2020, 25, 4085. doi:10.3390/molecules25184085 I...
Sunday, September 6, 2020

Registries for orphan drugs: generating evidence or marketing tools?

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Carla E. M. Hollak, Sandra Sirrs, Sibren van den Berg, Vincent van der Wel, Mirjam Langeveld, Hanka Dekker, Robin Lachmann & Saco J. de ...
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, ...