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 ...

MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich's Ataxia

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NEW YORK and GAINESVILLE, Fla., Jan. 13, 2020 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) and AavantiBio, a biotechnology compa...
Monday, January 13, 2020

Mammalian iron–sulfur cluster biogenesis: Recent insights into the roles of frataxin, acyl carrier protein and ATPase-mediated transfer to recipient proteins

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Nunziata Maio, Anshika Jain, Tracey A. Rouault; Current Opinion in Chemical Biology, Volume 55, 2020, Pages 34-44, doi:10.1016/j.cbpa.2019.1...
Friday, January 10, 2020

Retrotope Expands its Drug Pipeline with the First Dosing of RT001 in patients with Friedreich’s ataxia (FA)

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LOS ALTOS, Calif., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Retrotope announced today that it has dosed its first patient in a Phase 2/3 clinical t...
Thursday, January 9, 2020

Minoryx Therapeutics receives US FDA fast-track designation for leriglitazone in the treatment of X-ALD

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Minoryx Therapeutics. Press Releases. Mataró, Barcelona, Spain and Charleroi, Belgium, January 9, 2020 – Minoryx Therapeutics Leriglitazo...

Large-scale contractions of Friedreich’s ataxia GAA repeats in yeast occur during DNA replication due to their triplex-forming ability

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Alexandra N. Khristich, Jillian F. Armenia, Robert M. Matera, Anna A. Kolchinski, and Sergei M. Mirkin; PNAS first published January 7, 2020...
Wednesday, January 8, 2020

Multiple mechanisms underpin cerebral and cerebellar white matter deficits in Friedreich ataxia: The IMAGE‐FRDA study

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Louisa P. Selvadurai Louise A. Corben Martin B. Delatycki Elsdon Storey Gary F. Egan Nellie Georgiou‐Karistianis Ian H. Harding. Hum Brain M...
Tuesday, January 7, 2020

Heart-In-A-Jar Platform Humanizing Preclinical Research

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Bio-IT World.By Deborah Borfitz, January 6, 2020. The pumping capability was one of the more compelling features of the three-dimensional (...
Monday, January 6, 2020

Researchers suggest a pathway to reverse the genetic defect of Friedreich's ataxia

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Alexandra N. Khristich el al., "Large-scale contractions of Friedreich's ataxia GAA repeats in yeast occur during DNA replication d...
Friday, January 3, 2020

Longitudinal Increases in Cerebral Brain Activation During Working Memory Performance in Friedreich Ataxia: 24-Month Data from IMAGE-FRDA

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Rosita Shishegar, Ian H. Harding, Louise A. Corben, Martin B. Delatycki, Elsdon Storey, Gary F. Egan, Nellie Georgiou-Karistianis; Cerebellu...