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A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

• Source: Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants With MELAS (PRIZM)

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A Study to Assess TTI-0102 vs Placebo in MELAS Patients

• Source: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients With

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An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

• Source: An Open-label Extension Study Evaluating the Safety of Zagociguat in Participants With MELAS Who Completed TIS6463-203

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A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

• Authors : National Center for Advancing Translational Sciences (NCATS); Children's National Research Institute

• Source: Investigational Study of Glycerol Tributyrate in MELAS and LHON-Plus

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Pseudohypoxia-Stabilized HIF2α Transcriptionally Inhibits MNRR1 , a Druggable Target in MELAS.

• Authors : Purandare N; Center for Molecular Medicine and Genetics, School of Medicine, Wayne State University, Detroit, MI 48201, USA.; Pasupathi V

Subjects: Basic Helix-Loop-Helix Transcription Factors*/Basic Helix-Loop-Helix Transcription Factors*/Basic Helix-Loop-Helix Transcription Factors*/metabolism ; Basic Helix-Loop-Helix Transcription Factors*/Basic Helix-Loop-Helix Transcription Factors*/Basic Helix-Loop-Helix Transcription Factors*/genetics ; MELAS Syndrome*/MELAS Syndrome*/MELAS Syndrome*/genetics

• Source: Cells [Cells] 2025 Jul 15; Vol. 14 (14). Date of Electronic Publication: 2025 Jul 15.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409

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Multisystem clinicopathologic and genetic analysis of MELAS.

• Authors : Xu, Shuai¹ (AUTHOR) ; Jiang, Jialiu¹ (AUTHOR) ; Chang, Leilei¹ (AUTHOR)

• Source: Orphanet Journal of Rare Diseases. 12/24/2024, Vol. 19 Issue 1, p1-15. 15p.

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Cardiac manifestations in adult MELAS syndrome (mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes syndrome)– a cross-sectional study.

• Authors : Stoevesandt, Dietrich¹ (AUTHOR) ; Schlitt, Axel^2,3 (AUTHOR); Röntgen, Philipp¹ (AUTHOR)

• Source: Orphanet Journal of Rare Diseases. 2/10/2025, Vol. 20 Issue 1, p1-7. 7p.

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Cardiac Involvement in Patients with MELAS-Related mtDNA 3243A>G Variant.

• Authors : Vuorinen, Aino-Maija¹ (AUTHOR) ; Lehmonen, Lauri^1,2 (AUTHOR); Auranen, Mari^2,3 (AUTHOR)

• Source: Cardiogenetics. Jun2025, Vol. 15 Issue 2, p16. 8p.

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Signs, symptoms, and health-related quality of life in MELAS: measuring what’s important from the patient and clinician perspectives

• Authors : Paolo Medrano; Benjamin Banderas; Marisa Brimmer

Subjects: Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes; MELAS; Mitochondrial disease

• Source: Journal of Patient-Reported Outcomes, Vol 9, Iss 1, Pp 1-19 (2025)

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Stroke‐like episodes in patients with adult‐onset neuronal intranuclear inclusion disease and patients with late‐onset MELAS: A comparative study

• Authors : Yuzhi Shi; Gehong Dong; Hua Pan

Subjects: Male; Adult; Intranuclear Inclusion Bodies

• Source: Ann Clin Transl NeurolAnnals of Clinical and Translational Neurology, Vol 11, Iss 12, Pp 3125-3136 (2024)

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