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Related Concept Videos

Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

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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...
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Skeletal Muscle Relaxants: Therapeutic Uses01:31

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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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Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

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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...
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Disorders of the Skeletal Muscle01:28

Disorders of the Skeletal Muscle

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The clinical conditions affecting the skeletal muscle tissue are broadly categorized as musculoskeletal and neuromuscular disorders.
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
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Myasthenia Gravis: Diagnostic Tests01:15

Myasthenia Gravis: Diagnostic Tests

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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.
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Classification of Skeletal Muscle Relaxants01:28

Classification of Skeletal Muscle Relaxants

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Skeletal muscle relaxants are a group of drugs that can reduce muscle stiffness and induce temporary paralysis to relieve pain. These agents can act centrally to reduce muscle tone or spasms in painful conditions such as multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), or spinal injuries; they are called antispasmodics or spasmolytics.
Peripherally acting skeletal muscle relaxants interfere with the neurotransmission at the neuromuscular end plate to induce paralysis during...
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Related Experiment Video

Updated: Jun 23, 2025

Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice
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Clinical perspectives: Treating spinal muscular atrophy.

Molly A McPheron1, Marcia V Felker2

  • 1Department of Medical and Molecular Genetics, Indiana University, Indianapolis, IN 46202, USA.

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Spinal muscular atrophy (SMA) treatments like nusinersen, onasemnogene abeparvovec, and risdiplam offer hope. This review compares their evidence, administration, and future directions for this rare neuromuscular disease.

Keywords:
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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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Area of Science:

  • Neurology
  • Genetics
  • Pharmacology

Background:

  • Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disorder.
  • Untreated SMA leads to significant weakness and mortality.
  • Recent advancements have yielded three approved, high-cost treatments since 2016.

Purpose of the Study:

  • To review the evidence supporting current SMA treatments.
  • To discuss treatment selection, administration challenges, and monitoring.
  • To highlight the limited comparative data and future research needs.

Main Methods:

  • Literature review of studies on nusinersen, onasemnogene abeparvovec, and risdiplam.
  • Analysis of treatment efficacy, administration routes, and patient monitoring.
  • Synthesis of current data on comparative effectiveness and future therapeutic strategies.

Main Results:

  • Three effective treatments (nusinersen, onasemnogene abeparvovec, risdiplam) are available for SMA.
  • These treatments differ in administration methods and present unique challenges.
  • Direct comparative data between these therapies is limited.

Conclusions:

  • Current SMA treatments have transformed patient outcomes.
  • Treatment selection requires careful consideration of individual patient factors.
  • Further research is needed to directly compare therapies and optimize SMA management.