Thursday, December 31, 2020

Antisense Transcription across Nucleotide Repeat Expansions in Neurodegenerative and Neuromuscular Diseases: Progress and Mysteries

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Castro AF, Loureiro JR, Bessa J, Silveira I. Genes. 2020 Nov;11(12). DOI: 10.3390/genes11121418.

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich's Ataxia: Retrospective Study

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Mehmet Fatih YETKİN and Murat GÜLTEKİN; Noro Psikiyatr Ars. 2020 Sep 21;57(4):270-273. doi: 10.29399/npa.25047. eCollection 2020 Dec.   Inte...
Saturday, December 26, 2020

FDA requests new trial for Reata's Friedreich's ataxia program; J&J's Tremfya picks up expanded label in Europe

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Endpoints News, November 25, 2020. Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be...
Thursday, December 24, 2020

PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.

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NeurologyLive,  December 24, 2020 PTC Therapeutics has announced the initiation of the global phase 3 MOVE-FA study (NCT04577352) of vatiqui...

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia (MOVE-FA)

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ClinicalTrials.gov Identifier: NCT04577352; December 19, 2020  Sponsors and Collaborators  PTC Therapeutics   Double-blind, placebo-controll...

Insights Into the Roles of the Sideroflexins / SLC56 Family in Iron Homeostasis and Iron-Sulfur Biogenesis

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Tifoun, N.; De las Heras, J.M.; Guillaume, A.; Bouleau, S.; Mignotte, B.; Le Floch, N.; Preprints 2020, 2020120583 doi: 10.20944/preprints20...
Tuesday, December 22, 2020

Seelos Therapeutics Announces Issuance of a Patent for Trehalose (SLS-005) in Israel

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NEW YORK, Dec. 21, 2020 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the ...
Friday, December 18, 2020

Rehabilitation for ataxia study: protocol for a randomised controlled trial of an outpatient and supported home-based physiotherapy programme for people with hereditary cerebellar ataxia

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Sarah C Milne1, Louise A Corben, Melissa Roberts, David Szmulewicz, J Burns, Anneke C Grobler, Shannon Williams, Jillian Chua, Christina Lia...

Effects of Fe2+/Fe3+ Binding to Human Frataxin and Its D122Y Variant, as Revealed by Site-Directed Spin Labeling (SDSL) EPR Complemented by Fluorescence and Circular Dichroism Spectroscopies

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Doni, D.; Passerini, L.; Audran, G.; Marque, S.R.A.; Schulz, M.; Santos, J.; Costantini, P.; Bortolus, M.; Carbonera, Int. J. Mol. Sci. 2020...
Wednesday, December 16, 2020

Minoryx’s clinical candidate leriglitazone shows clinical benefit in a proof of concept Phase 2 study in Friedreich´s ataxia

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Mataró, Barcelona, Spain, December 15, 2020 - Minoryx Therapeutics, a Phase 3 clinical stage biotech company focused on the development of d...