Thursday, November 3, 2022

Design Therapeutics Highlights Pipeline Progress and Upcoming Milestones and Reports Third Quarter 2022 Financial Results

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CARLSBAD, Calif., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company develop...

Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia, A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES)

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Massimo Pandolfo, Kathrin Reetz, Alejandra Darling, Francisco Javier Rodriguez de Rivera, Pierre-Gilles Henry, James Joers, Christophe Lengl...
Sunday, October 30, 2022

Progressive Spinal Cord Degeneration in Friedreich's Ataxia: Results from ENIGMA-Ataxia

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Thiago J.R. Rezende PhD, Isaac M. Adanyeguh PhD, Filippo Arrigoni MD, Benjamin Bender MD, Fernando Cendes MD, PhD, Louise A. Corben PhD, And...
Saturday, October 22, 2022

Spinal cord magnetic resonance imaging and spectroscopy detect early-stage alterations and disease progression in Friedreich ataxia

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James M Joers, Isaac M Adanyeguh, Dinesh K Deelchand, Diane H Hutter, Lynn E Eberly, Isabelle Iltis, Khalaf O Bushara, Christophe Lenglet, P...
Thursday, October 20, 2022

Larimar Therapeutics Announces Issuance of U.S. Patent Providing Composition of Matter Protection for CTI-1601

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BALA CYNWYD, Pa., Oct. 20, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology co...
Tuesday, October 18, 2022

Children with a rare congenital genetic disorder: a systematic review of parent experiences

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von der Lippe, C., Neteland, I. & Feragen, K.B.; Orphanet J Rare Dis 17, 375 (2022). doi:10.1186/s13023-022-02525-0   Coordinated care,...
Monday, October 17, 2022

Selection of Synthetic Proteins to Modulate the Human Frataxin Function

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Pignataro MF, Herrera MG, Fernández N, Aran M, Gentili H, Bataglini F, Santos J.; Biotechnol Bioeng. 2022 Oct 12. doi: 10.1002/bit.28263. Ep...
Friday, October 14, 2022

Reata Pharmaceuticals Announces that the FDA Does Not Plan to Hold an Advisory Committee Meeting to Discuss the NDA for Omaveloxolone for Friedreich’s Ataxia

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October 13, 2022. PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”),...
Thursday, October 13, 2022

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

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ClinicalTrials.gov Identifier: NCT05573698. A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose...
Wednesday, October 12, 2022

SARS-CoV-2 in patients with Friedreich ataxia

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Megan M. Shen, Layne N. Rodden, Kellie McIntyre, Adriana Arias, Victoria Profeta, Kimberly Schadt & David R. Lynch; J Neurol (2022). do...