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220 results on '"Myotonic Dystrophy drug therapy"'

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1. The AMPK allosteric activator MK-8722 improves the histology and spliceopathy in myotonic dystrophy type 1 (DM1) skeletal muscle.

2. AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action.

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3. Expert opinion on mexiletine treatment in adult patients with myotonic dystrophy.

4. Therapeutic potential of oleic acid supplementation in myotonic dystrophy muscle cell models.

5. Efficacy of methylphenidate treatment in childhood myotonic dystrophy type 1 and comorbid attention deficit hyperactivity disorder: A case report using eye tracking assessment.

6. Verapamil mitigates chloride and calcium bi-channelopathy in a myotonic dystrophy mouse model.

7. Expert Insights from a Delphi-driven Neurologists' Panel: Real-world Mexiletine use in Patients with Myotonic Disorders in Italy.

8. Clearance of defective muscle stem cells by senolytics restores myogenesis in myotonic dystrophy type 1.

9. Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy.

10. Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSA LR Mice.

11. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.

12. The myotonic dystrophy type 1 drug development pipeline: 2022 edition.

13. Impact of restricted access to, and low awareness of, mexiletine on people with myotonia: a real-world European survey.

14. Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.

15. Combinatorial treatment with exercise and AICAR potentiates the rescue of myotonic dystrophy type 1 mouse muscles in a sex-specific manner.

16. Repurposing pentamidine using hyaluronic acid-based nanocarriers for skeletal muscle treatment in myotonic dystrophy.

17. Calcitriol increases MBNL1 expression and alleviates myotonic dystrophy phenotypes in HSA LR mouse models.

18. Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1.

19. Recent Progress and Challenges in the Development of Antisense Therapies for Myotonic Dystrophy Type 1.

21. A patent and literature review of CDK12 inhibitors.

22. Selective and Reversible Ligand Assembly on the DNA and RNA Repeat Sequences in Myotonic Dystrophy.

23. Pharmacological and exercise-induced activation of AMPK as emerging therapies for myotonic dystrophy type 1 patients.

24. Pharmacotherapy alleviates pathological changes in human direct reprogrammed neuronal cell model of myotonic dystrophy type 1.

25. A Selective Alkylating Agent for CTG Repeats in Myotonic Dystrophy Type 1.

26. Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing.

27. Targeting Myotonic Dystrophy Type 1 with Metformin.

28. [Effect of Small Molecules on Repeat RNA Toxicity in Animal Models].

29. Aurintricarboxylic Acid Decreases RNA Toxicity in a C. elegans Model of Repeat Expansions.

31. A Novel Minor Groove Binder as a Potential Therapeutic Agent for Myotonic Dystrophy Type 1.

32. Myotonic dystrophy type 1 drug development: A pipeline toward the market.

33. Repeat RNA expansion disorders of the nervous system: post-transcriptional mechanisms and therapeutic strategies.

34. A Small Molecule that Binds an RNA Repeat Expansion Stimulates Its Decay via the Exosome Complex.

35. Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial.

36. The Dimeric Form of 1,3-Diaminoisoquinoline Derivative Rescued the Mis-splicing of Atp2a1 and Clcn1 Genes in Myotonic Dystrophy Type 1 Mouse Model.

37. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1).

38. Expanded DNA and RNA Trinucleotide Repeats in Myotonic Dystrophy Type 1 Select Their Own Multitarget, Sequence-Selective Inhibitors.

39. Learnings from Patient-Report Workshop on Disease Progression in Myotonic Dystrophy.

40. Protective effects of mirtazapine in mice lacking the Mbnl2 gene in forebrain glutamatergic neurons: Relevance for myotonic dystrophy 1.

41. The Association of methylprednisolone dosing to cessation of myotonia in a patient with myotonic dystrophy type 1.

42. CDK12 inhibition reduces abnormalities in cells from patients with myotonic dystrophy and in a mouse model.

43. Myotonic Dystrophy type 1 cells display impaired metabolism and mitochondrial dysfunction that are reversed by metformin.

45. Inhibition of cyclooxygenase-1 by nonsteroidal anti-inflammatory drugs demethylates MeR2 enhancer and promotes Mbnl1 transcription in myogenic cells.

46. Biophysical mechanisms for QRS- and QTc-interval prolongation in mice with cardiac expression of expanded CUG-repeat RNA.

47. Correction of RNA-Binding Protein CUGBP1 and GSK3β Signaling as Therapeutic Approach for Congenital and Adult Myotonic Dystrophy Type 1.

48. Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models.

49. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.

50. Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules.