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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...
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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.
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Skeletal muscle relaxants are widely used for muscle paralysis and relieving pain following any muscle injury or stiffness. However, depending on the drug type, they can have adverse effects that range from mild to severe. Usually, nondepolarizing neuromuscular blockers have minimal side effects. For example, drugs like d-tubocurarine, cisatracurium, and rocuronium cause hypotension, whereas drugs like baclofen, when stopped abruptly, can lead to the recurrence of spastic conditions.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Updated: Oct 14, 2025

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Risdiplam for the Use of Spinal Muscular Atrophy.

Juyeon Kakazu1, Nakoma L Walker2, Katherine Claire Babin2

  • 1Georgetown University School of Medicine, Washington DC.

Orthopedic Reviews
|November 8, 2021
PubMed
Summary
This summary is machine-generated.

Spinal muscular atrophy (SMA) is a leading infant death cause. Risdiplam, an oral medication, offers a novel treatment by increasing survival motor neuron protein (SMN) expression, overcoming limitations of prior therapies.

Keywords:
evrysdinusinersenrisdiplamsmaspinal muscular atrophy

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Area of Science:

  • Neurology
  • Genetics
  • Pharmacology

Background:

  • Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disorder characterized by motor neuron degeneration.
  • SMA leads to progressive muscle weakness and atrophy, significantly impacting infant mortality.
  • Current treatments aim to increase survival motor neuron (SMN) protein levels, crucial for motor neuron function.

Purpose of the Study:

  • To review the background, clinical studies, and indications for Risdiplam in treating SMA.
  • To highlight Risdiplam's role as a novel therapeutic agent for SMA.
  • To compare Risdiplam with existing SMA treatment modalities.

Main Methods:

  • Review of existing literature on SMA and its treatments.
  • Analysis of clinical studies investigating Risdiplam's efficacy and safety.
  • Comparison of Risdiplam's mechanism of action and administration route with other SMA therapies.

Main Results:

  • Risdiplam is the first FDA-approved oral medication for SMA.
  • It functions as an SMN2 splicing modifier, enhancing SMN protein expression systemically.
  • Risdiplam offers advantages over intrathecal and gene replacement therapies, including non-invasive administration and broader systemic reach.

Conclusions:

  • Risdiplam represents a significant advancement in SMA treatment, offering an accessible oral option.
  • Its systemic effects and non-invasive nature are beneficial, particularly for infants.
  • Further research into Risdiplam's safety profile compared to other therapies is warranted due to its systemic action.