Monday, May 15, 2023

Larimar Therapeutics Reports Preliminary Top-line Data from Phase 2 Trial’s 25 mg Cohort Showing Increases in Frataxin Levels in Patients with Friedreich’s Ataxia

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BALA CYNWYD, Pa., May 15, 2023 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology com...

New Drug Evaluation: Omaveloxolone Oral Capsules

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Drug Use Research & Management Program. Oregon State University. Date of Review: June 2023. Brand Name (Manufacturer): Skyclarys™ (Reata...
Friday, May 12, 2023

Frataxin deficiency disrupts mitochondrial respiration and pulmonary endothelial cell function

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Culley MK, Rao RJ, Mehta M, Zhao J, El Khoury W, Harvey LD, Perk D, Tai YY, Tang Y, Shiva S, Rabinovitch M, Gu M, Bertero T, Chan SY. Fratax...

AIDE À LA PRISE EN CHARGE DU PATIENT ATTEINT D'UNE MALADIE RARE : l'ataxie de Friedreich

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Iris Marolleau. Kinésithér Scient 2023,0653:15-20 - 10/05/2023 L'ataxie de Friedreich est une maladie rare du système nerveux central. C...

Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

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Dr. Carlos Munoz-Zuluaga, Dr. Monica Gertz, Ms. Melissa Yost-Bido, Ms. Alessandria Greco, Mr. Nicholas Gorman, Mr. Alvin Chen, Mr. Vikrum Ko...
Wednesday, May 10, 2023

Design Therapeutics Provides Pipeline Updates and Reports First Quarter 2023 Financial Results

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CARLSBAD, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.  Initial Data from Ongoing Phase 1 MAD Trial of DT-216 for FA E...
Tuesday, May 9, 2023

Omaveloxolone: First Approval

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Lee A. Omaveloxolone: First Approval. Drugs. 2023 May 8. doi: 10.1007/s40265-023-01874-9. Epub ahead of print. PMID: 37155124.  Omaveloxolon...
Monday, May 8, 2023

Omaveloxolone (SkyclarysTM) for patients with Friedreich’s ataxia

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Sharadha Dayalan Naidu, Albena T. Dinkova-Kostova, Omaveloxolone (SkyclarysTM) for patients with Friedreich’s ataxia, Trends in Pharmacologi...
Saturday, May 6, 2023

A systematic overview of rare disease patient registries: challenges in design, quality management, and maintenance

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Hageman, I.C., van Rooij, I.A., de Blaauw, I. et al. A systematic overview of rare disease patient registries: challenges in design, quality...
Wednesday, May 3, 2023

Ketolysis is required for the proper development and function of the somatosensory nervous system

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Enders J, Jack J, Thomas S, Lynch P, Lasnier S, Cao X, Swanson MT, Ryals JM, Thyfault JP, Puchalska P, Crawford PA, Wright DE. Ketolysis is ...