Thursday, August 10, 2023

Omaveloxolone for the Treatment of Friedreich’s Ataxia

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Riley Kessler, Sonal Sharma, David R Lynch, Omaveloxolone for the Treatment of Friedreich’s Ataxia, Published Online: Aug 9th 2023 touchREVI...
Monday, August 7, 2023

CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective

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Sahel DK, Vora LK, Saraswat A, et al. CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Pers...

Preparing for Disease-Modification Trials in Degenerative Cerebellar Ataxias: Which Endpoints to Choose?

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Maas RPPWM. Preparing for Disease-Modification Trials in Degenerative Cerebellar Ataxias: Which Endpoints to Choose? Movement Disorders : Of...

Brain MRI detects early-stage alterations and disease progression in Friedreich ataxia

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Adanyeguh IM, Joers JM, Deelchand DK, et al. Brain MRI detects early-stage alterations and disease progression in Friedreich ataxia. Brain C...
Sunday, August 6, 2023

Recent advances in small molecules for improving mitochondrial disorders

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Meng L, Wu G. Recent advances in small molecules for improving mitochondrial disorders. RSC Advances. 2023 Jul;13(30):20476-20485. DOI: 10.1...
Saturday, August 5, 2023

Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app

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Buchholz M, Weber N, Borel S, et alPatient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): p...
Thursday, August 3, 2023

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

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Poleur, M., Markati, T. & Servais, L. The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic...
Friday, July 28, 2023

Biogen to Acquire Reata Pharmaceuticals

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CAMBRIDGE, Mass. and PLANO, Texas, July 28, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Reata Pharmaceuticals, Inc. (Nasdaq: RET...
Tuesday, July 25, 2023

Larimar Therapeutics Receives FDA Clearance to Proceed to 50 mg Cohort in CTI-1601’s Phase 2 Friedreich's Ataxia Trial and to Initiate Open Label Extension Trial

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BALA CYNWYD, Pa., July 25, 2023 (GLOBE NEWSWIRE). Larimar Therapeutics, Inc.. Today announced that the U.S. Food and Drug Administration (FD...
Monday, July 24, 2023

Leriglitazone

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National Center for Biotechnology Information. PubChem Compound Summary for CID 4147757, Hydroxypioglitazone. https://pubchem.ncbi.nlm.nih.g...