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Related Concept Videos

Directly Acting Muscle Relaxants: Dantrolene and Botulinum Toxin01:26

Directly Acting Muscle Relaxants: Dantrolene and Botulinum Toxin

Directly acting muscle relaxants like dantrolene and botulinum toxin (BoNT) have distinct mechanisms and applications. Dantrolene, a hydantoin derivative, acts on the ryanodine receptor (RYR1) in skeletal muscle cells. RYR1 are calcium channels present at the sarcoplasmic reticulum membrane. In response to excitation, they release calcium ions from the sarcoplasmic reticulum to the cytosol. Calcium promotes actin-myosin-mediated contraction of muscles.
The binding of dantrolene to the RYR1...
Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

Myasthenia gravis is a neuromuscular transmission disorder characterized by weakness and increased fatigability of skeletal muscles. It is an autoimmune disease affecting approximately one in 2000 people, where antibodies against the α1 subunit of nicotinic acetylcholine receptors are produced.
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Formation of Muscle Fibers from Myoblasts01:13

Formation of Muscle Fibers from Myoblasts

De novo myogenesis, or the formation of muscle fibers, begins during the early embryonic stages. The skeletal muscle is formed from somites– blocks of embryonic cell layers. The somites are further divided into dermatomes, myotomes, sclerotomes, and syndetomes. Among these, the myotomes give rise to muscle fibers.
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Skeletal Muscle Relaxants: Therapeutic Uses

Skeletal muscle relaxants are used to relax muscle tone and alleviate painful muscle contractions. However, the choice of skeletal muscle relaxants depends on the duration of the surgical procedure in order to minimize potential side effects. Skeletal muscle relaxants like neuromuscular blocking agents [NMBAs] are commonly employed as adjuvants alongside general anesthetics in clinical settings. NMBAs are also used to maintain controlled ventilation during surgery of the larynx or pharynx as...
Myasthenia Gravis ll: Pathophysiology01:22

Myasthenia Gravis ll: Pathophysiology

The disease process of myasthenia gravis begins at the neuromuscular junction, where antibodies attack key proteins needed for muscle activation. This immune reaction weakens signal transmission, leading to the characteristic muscle fatigue and weakness that define the condition.Immune-Mediated DamageIn most individuals, antibodies target acetylcholine receptors (AChRs) on the postsynaptic membrane of muscle cells. By blocking acetylcholine binding, these antibodies prevent the nerve signal...

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Oleic Acid Levels in HSA<sup>LR</sup> Mouse Model of Myotonic Dystrophy Type 1.

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Updated: May 31, 2026

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
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Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

The myotonic dystrophy type 1 drug development pipeline: 2026 Edition.

Yasmine Ferchichi1, Àngela Natividad1, Rubén Artero1

  • 1Human Translational Genomics Group, University Institute for Biotechnology and Biomedicine (BIOTECMED), University of Valencia, Av. Vicent Andrés Estellés, 19, Valencia 46100, Spain; INCLIVA Biomedical Research Institute, Av. Menendez Pelayo 4, Valencia 46010, Spain; Ciberer Isciii, Av. Monforte de Lemos 3-5, Madrid 28029, Spain.

Drug Discovery Today
|May 28, 2026
PubMed
Summary

Therapeutic development for myotonic dystrophy type 1 is rapidly advancing with diverse strategies like gene therapy and antisense platforms. Promising nucleic-acid-based treatments show potential for disease modification, alongside rehabilitation interventions.

Keywords:
CTG repeat expansionantisense oligonucleotidesclinical trialsdrug repurposinggene therapymyotonic dystrophynucleic acid therapeutics

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Assessing Functional Performance in the Mdx Mouse Model

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Related Experiment Videos

Last Updated: May 31, 2026

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
09:39

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia
10:41

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia

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Assessing Functional Performance in the Mdx Mouse Model
10:32

Assessing Functional Performance in the Mdx Mouse Model

Published on: March 27, 2014

Area of Science:

  • Biomedical research
  • Translational medicine
  • Neurology

Background:

  • Myotonic dystrophy type 1 (DM1) is a progressive genetic disorder.
  • Therapeutic development for DM1 has historically faced challenges.
  • Recent advancements signal a shift towards effective treatments.

Purpose of the Study:

  • To review the current landscape of therapeutic development for DM1.
  • To highlight emerging treatment modalities and their progress.
  • To assess the potential for clinical translation of novel therapies.

Main Methods:

  • Analysis of ongoing clinical trials and preclinical research in DM1.
  • Review of diverse therapeutic approaches including small molecules, gene therapy, and nucleic-acid-based strategies.
  • Evaluation of non-pharmacological interventions like structured exercise.

Main Results:

  • Significant acceleration in DM1 therapeutic development, with a doubling of clinical trials.
  • Advancement of multiple candidates, particularly nucleic-acid-based therapies, to late-stage evaluation.
  • Demonstration of robust target engagement and clinically meaningful signals from nucleic-acid therapies.

Conclusions:

  • The therapeutic landscape for DM1 is maturing rapidly with multiple promising avenues.
  • Nucleic-acid-based therapies are highly promising for DM1 disease modification.
  • A combination of pharmacological and non-pharmacological interventions may offer comprehensive disease management.