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The Study of the Inheritance Mechanisms of Myotonic Dystrophy Type 1 (DM1) in Families from the Republic of North Ossetia-Alania.

• Authors : Ionova, Sofya A.¹ (AUTHOR) ; Murtazina, Aysylu F.¹ (AUTHOR) ; Marakhonov, Andrey A.¹ (AUTHOR)

Subjects: *MUSCLE weakness; *MYOTONIA atrophica; *SYMPTOMSNORTH Ossetia (Russia)

• Source: International Journal of Molecular Sciences. Sep2024, Vol. 25 Issue 17, p9734. 11p.

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A Clinical Study of PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)

• Source: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic

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Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (ACHIEVE)

• Source: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With

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Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1 (FREEDOM-DM1)

• Source: A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult

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Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)

• Source: A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult

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Disturbance of the human gut microbiota in patients with Myotonic Dystrophy type 1

• Authors : Manijeh Mahdavi; Karine Prévost; Philippe Balthazar

Subjects: Myotonic Dystrophy type 1; DM1; Microbiome

• Source: Computational and Structural Biotechnology Journal, Vol 23, Iss , Pp 2097-2108 (2024)

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Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

• Authors : University of Rochester; The Methodist Hospital Research Institute; University of Kansas

• Source: Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1

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Venous Thromboembolism in Myotonic Dystrophy Type 1 (DM1-VTE)

• Authors : AFM-Téléthon (Funding); Recherche Clinique Paris Descartes Necker Cochin Sainte Anne

• Source: Venous Thromboembolism in Myotonic Dystrophy Type 1Udd B, Krahe R. The

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The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2

• Authors : Lupin Atlantis Holdings S.A.

• Source: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With

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Latent factors underlying the symptoms of adult-onset myotonic dystrophy type 1 during the clinical course.

• Authors : Zhang, Yanan¹ (AUTHOR); Wallace, Bailey^2,3 (AUTHOR); Cai, Bo¹ (AUTHOR)

• Source: Orphanet Journal of Rare Diseases. 11/1/2024, Vol. 19 Issue 1, p1-10. 10p.

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