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 ...

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...