Saturday, July 16, 2022

SCouT: Synthetic Counterfactuals via Spatiotemporal Transformers for Actionable Healthcare

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Bhishma Dedhia, Roshini Balasubramanian, Niraj K. Jha; arXiv:2207.04208v1 [cs.AI] for this version); doi:10.48550/arXiv.2207.04208 (Computer...
Thursday, July 14, 2022

Cardiovascular Research in Friedreich Ataxia

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R. Mark Payne; J Am Coll Cardiol Basic Trans Science. null2022, 0 (0); doi:10.1016/j.jacbts.2022.04.005 Patients can develop a cardiomyopath...

Cerebrospinal Fluid Proteomics in Friedreich Ataxia Reveals Markers of Neurodegeneration and Neuroinflammation

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Imbault, Virginie; Dionisi, Chiara; Naeije, Gilles; Communi, David; Pandolfo, Massimo (2022). Frontiers. Collection. doi:10.3389/fnins.2022....
Wednesday, July 13, 2022

EE146 Cost and Resource Utilization in Friedreich Ataxia: A Systematic Literature Review

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R Zhang, K Buesch; Value in Health, Volume 25, Issue 7, Supplement, 2022, Page S363, doi:10.1016/j.jval.2022.04.396.  The search retrieved 5...

CO49 Clinical Efficacy and Safety of Therapeutic Interventions Used in Friedreich Ataxia: A Systematic Review

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P Jain, L Badgujar, JA Spoorendonk, K Buesch; Value in Health, Volume 25, Issue 7, Supplement, 2022, Pages S312-S313, doi:10.1016/j.jval.20...

Natural History of Friedreich's Ataxia: Heterogeneity of Neurological Progression and Consequences for Clinical Trial Design

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Christian Rummey, Louise A Corben, Martin Delatycki, George Wilmot, Sub H Subramony, Manuela Corti, Khalaf Bushara, Antoine Duquette, Christ...
Tuesday, July 12, 2022

Rare motor disorder: International study investigates living situation of people with Friedreich’s Ataxia

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AAAS and EurekAlert. NEWS RELEASE 11-JUL-2022 DZNE - GERMAN CENTER FOR NEURODEGENERATIVE DISEASES  The study aims to investigate and evaluat...
Monday, July 11, 2022

셀리버리 “올해 코로나19·아토피 2상 진입 기대…무증 적절”

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[KBIC 2022] 조대웅 대표 발표  프리드리히 운동실조증(FRDA) 치료제 ‘AAV-FXN-aMTD‘는 TSDT를 적용한 세포·조직투과성 aMTD-융합 Frataxin(FXN) 재조합단백질이다. 조 대표는 “현재 다케다 제약과 공동 연구개발을 진...
Sunday, July 10, 2022

HTA decision-making for drugs for rare diseases: comparison of processes across countries

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Tania Stafinski, Judith Glennie, Andrea Young & Devidas Menon; Orphanet J Rare Dis 17, 258 (2022). doi:10.1186/s13023-022-02397-4  There...
Thursday, July 7, 2022

A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT05445323; Sponsor: Lexeo Therapeutics Intervention/treatment:  Genetic: Low dose LX2006 Adeno-associated ...