Thursday, May 25, 2023

A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia

›
Subramony SH, Lynch DL. A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia. Cerebellum (London, Englan...

Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia (CPCAF)

›
ClinicalTrials.gov Identifier: NCT05874388. First Posted: May 24, 2023. Sponsors and Collaborators: Institut National de la Santé Et de la R...
Wednesday, May 24, 2023

PTC Therapeutics Discontinues Some Programs In Gene Therapy

›
May 23, 2023. (RTTNews) - PTC Therapeutics Inc. (PTCT) said that it has discontinued pre-clinical and early research programs in gene therap...
Tuesday, May 23, 2023

PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial Twitter Facebook LinkedIn GooglePlus Pinterest

›
SOUTH PLAINFIELD, N.J. , May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA ...

Large-scale expansions of Friedreich's ataxia GAA•TTC repeats in an experimental human system: role of DNA replication and prevention by LNA-DNA oligonucleotides and PNA oligomers

›
Anastasia Rastokina, Jorge Cebrián, Negin Mozafari, Nicholas H Mandel, C I Edvard Smith, Massimo Lopes, Rula Zain, Sergei M Mirkin, Large-sc...
Monday, May 22, 2023

The Loss of Frataxin Impairs Microglia Homeostatic Functions in Friedreich’s Ataxia

›
Ferrara G. Abstracts of the Fifth Brainstorming Research Assembly for Young Neuroscientists (BraYn), Italy, 28-30 September 2022. Neurology ...
Sunday, May 21, 2023

Neuropsychiatric symptoms in spinocerebellar ataxias and Friedreich ataxia

›
Karamazovova S, Matuskova V, Ismail Z, Vyhnalek M. Neuropsychiatric symptoms in spinocerebellar ataxias and Friedreich ataxia. Neuroscience ...
Wednesday, May 17, 2023

Patient-Derived iPSC-Neurons and Microglia for Modeling Friedreich’s Ataxia Disease

›
1694 Patient-Derived iPSC-Neurons and Microglia for Modeling Friedreich’s Ataxia Disease; Priyanka Mishra, Anusha Sivakumar, Avalon Johnson,...

Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia

›
1656 Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia; Jonathan B. Rosenberg, Alessandri...

Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications

›
1549 Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications; Jennifer C. G. Green, Jessica F. Boehl...