Tuesday, April 2, 2024

Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study

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Rummey C, Perlman S, Subramony SH, Farmer J, Lynch DR. Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD stud...
Friday, March 29, 2024

Therapeutic Potential of Dimethyl Fumarate to Treat Friedreich Ataxia: Francesco Saccà, MD, PhD

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NeurologyLive. March 28, 2024. Fracesco Saccà, MD, PhD In an interview with NeurologyLive®, Saccà discussed the idea behind the study and th...
Monday, March 25, 2024

Novel approach for evaluation of mitochondrial substrate utilization in fibroblasts from patients with Friedreich’s ataxia

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Ahmadian N, Dallas S, Dedkova EN. Novel approach for evaluation of mitochondrial substrate utilization in fibroblasts from patients with Fri...

Using synthetic genome readers/regulators to interrogate chromatin processes: A brief review

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Philips SJ, Danda A, Ansari AZ. Using synthetic genome readers/regulators to interrogate chromatin processes: A brief review. Methods. 1 de ...

Unique combinations of ultrasound and electrophysiological findings distinguish Friedreich’s ataxia from other inherited ataxias

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Pelosi L, Mulroy E, Leadbetter R, Rodrigues M, Roxburgh R. Unique combinations of ultrasound and electrophysiological findings distinguish F...

Larimar Triumphs: Significant Strides Made on the Road to Treating Friedreich’s Ataxia

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BALA CYNWYD, PA — Larimar Therapeutics, Inc. March 19, 2024   In their ongoing discussions with the FDA, Larimar is working on a potential u...

Design Therapeutics, Inc. (NASDAQ:DSGN) Q4 2023 Earnings Call Transcript

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Published on March 19 on Seeking Alpha. Transcripts. We learned from the human studies that the duration of adequate levels of exposure of D...
Wednesday, March 20, 2024

Stealth BioTherapeutics Presents Data of Novel Compound, SBT-589, in Friedreich's Ataxia Cardiac Models at the Wellcome Trust Mitochondrial Medicine Conference

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NEEDHAM, Mass., March 19, 2024 /PRNewswire/ -- Stealth Biotherapeutics Inc.  SBT-589 showed mitochondrial protection across models of Friedr...
Tuesday, March 19, 2024

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

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CARLSBAD, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc..  New Drug Product for Friedreich Ataxia (FA) DT-216P2 with F...

Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity

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Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity. Front. Pharmacol., 19 March 2024, Sec. Neurop...