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Related Concept Videos

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.
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which leads...
Myasthenia Gravis: Diagnostic Tests01:15

Myasthenia Gravis: Diagnostic Tests

Myasthenia gravis is an autoimmune condition affecting neuromuscular transmission, causing generalized weakness in skeletal muscles. Initial diagnoses rely on patients' signs, symptoms, and medical history. The challenge lies in distinguishing myasthenia from other muscular dystrophies. An important diagnostic feature is the significant improvement of symptoms after administering anticholinesterase inhibitors.
The edrophonium test is a diagnostic tool for myasthenia gravis. It involves...
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...
Nondepolarizing (Competitive) Neuromuscular Blockers: Mechanism of Action01:17

Nondepolarizing (Competitive) Neuromuscular Blockers: Mechanism of Action

Nondepolarizing neuromuscular blockers induce paralysis by competitively blocking nicotinic acetylcholine receptors at the muscle end plate. Examples include pancuronium, mivacurium, vecuronium, and rocuronium. These quaternary ammonium derivatives are administered intravenously, are poorly absorbed, and are excreted via the kidneys.
Competitive antagonists prevent acetylcholine from binding to its receptor, inhibiting membrane depolarization. Without conformational changes or intrinsic...
Chemical Synapses01:26

Chemical Synapses

Chemical synapses are specialized sites between two neurons or between a neuron and a non-neuronal cell like a muscle, glandular or sensory cell.
Because chemical synapses depend on the release of neurotransmitter molecules from synaptic vesicles to pass on their signal, there is an approximately one millisecond delay between when the axon potential reaches the presynaptic terminal and when the neurotransmitter leads to opening of postsynaptic ion channels. Additionally, this signaling is...
Chemical Synapses01:26

Chemical Synapses

Chemical synapses are specialized sites between two neurons or between a neuron and a non-neuronal cell like a muscle, glandular or sensory cell.
Because chemical synapses depend on the release of neurotransmitter molecules from synaptic vesicles to pass on their signal, there is an approximately one millisecond delay between when the axon potential reaches the presynaptic terminal and when the neurotransmitter leads to opening of postsynaptic ion channels. Additionally, this signaling is...

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Related Experiment Videos

Another complement inhibitor therapy for myasthenia gravis: Is it NIMBLE enough?

Linda L Kusner1, Henry J Kaminski2

  • 1Laboratory for Myasthenia Gravis Research, George Washington University, Washington, DC, USA; Department of Pharmacology & Physiology, George Washington University, Washington, DC, USA.

Med (New York, N.Y.)
|July 10, 2026
PubMed
Summary

The NIMBLE trial shows cemdisiran, an siRNA inhibitor, is effective for myasthenia gravis. This novel complement inhibitor expands treatment options for this neuromuscular disease.

Related Experiment Videos

Area of Science:

  • Neurology
  • Immunology
  • Pharmacology

Background:

  • Myasthenia gravis is a rare autoimmune disorder affecting neuromuscular junctions.
  • Current treatments for myasthenia gravis include immunosuppressants and FDA-approved complement inhibitors.
  • There is a need for novel therapeutic mechanisms to address complement-mediated pathways in myasthenia gravis.

Purpose of the Study:

  • To evaluate the efficacy of cemdisiran, a novel siRNA inhibitor of hepatic C5 synthesis, in patients with myasthenia gravis.

Main Methods:

  • The study was a phase 3 clinical trial named NIMBLE.
  • Participants received cemdisiran, targeting hepatic C5 synthesis.

Main Results:

  • The NIMBLE trial demonstrated the efficacy of cemdisiran in patients with myasthenia gravis.
  • Cemdisiran represents a first-in-class complement inhibitor for myasthenia gravis utilizing this specific mechanism.

Conclusions:

  • Cemdisiran shows promise as a new therapeutic option for myasthenia gravis.
  • The findings contribute to the expanding treatment landscape for myasthenia gravis, particularly for complement-mediated forms.