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Pelizaeus-Merzbacher Disease Medication

  • Author: Jasvinder Chawla, MD, MBA; Chief Editor: Selim R Benbadis, MD  more...
 
Updated: Jul 11, 2016
 

Medication Summary

No specific medications are available for treatment of Pelizaeus-Merzbacher disease. However, some patients may benefit from antispasticity medications, such as baclofen (including intrathecally administered baclofen), tizanidine, and benzodiazepines. Botulinum toxin injections in spastic muscles or salivary glands can be very helpful in managing spasticity or sialorrhea/drooling, respectively. Children with seizures need to be appropriately treated.

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Anxiolytics, Benzodiazepines

Class Summary

These agents may potentiate the effects of gamma-aminobutyric acid (GABA) and facilitate inhibitory GABA neurotransmission.

Diazepam (Valium, Diastat)

 

Diazepam is useful in suppressing muscle contractions by facilitating inhibitory GABA neurotransmission and other inhibitory transmitters.

Lorazepam (Ativan)

 

Lorazepam is a sedative-hypnotic of the benzodiazepine class that has a rapid onset of effect and a relatively long half-life. By increasing the action of gamma-aminobutyric acid (GABA), a major inhibitory neurotransmitter, it may depress all levels of the central nervous system (CNS), including the limbic system and reticular formations. Lorazepam is excellent for patients who need to be sedated for longer than 24 hours.

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Skeletal Muscle Relaxants

Class Summary

These agents may inhibit the transmission of monosynaptic and polysynaptic reflexes at the spinal cord level.

Baclofen (Lioresal, Gablofen)

 

Baclofen may induce hyperpolarization of afferent terminals and inhibit monosynaptic and polysynaptic reflexes at the spinal level.

Cyclobenzaprine (Flexeril, Fexmid, Amrix)

 

Cyclobenzaprine acts centrally and reduces motor activity of tonic somatic origins, influencing alpha and gamma motor neurons. It is structurally related to the tricyclic antidepressants.

Skeletal muscle relaxants have modest, short-term benefit as adjunctive therapy for nociceptive pain associated with muscle strains and, used intermittently, for diffuse and certain regional chronic pain syndromes. Long-term improvement over placebo has not been established.

Cyclobenzaprine often produces a "hangover" effect, which can be minimized by taking the nighttime dose 2-3 hours before going to sleep.

Carisoprodol (Soma)

 

Carisoprodol is a short-acting medication that may have depressant effects at the spinal cord level.

Skeletal muscle relaxants have modest short-term benefit as adjunctive therapy for nociceptive pain associated with muscle strains and, used intermittently, for diffuse and certain regional chronic pain syndromes. Long-term improvement over placebo has not been established.

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Alpha2-adrenergic Agonist Agents

Class Summary

These agents have beneficial antispasticity effects.

Tizanidine (Zanaflex)

 

Tizanidine is a centrally acting muscle relaxant that is metabolized in the liver and excreted in the urine and feces.

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Neuromuscular Blockers, Botulinum Toxins

Class Summary

Agents in this class cause presynaptic paralysis of the myoneural junction and reduce abnormal contractions.

Botulinum toxin (BOTOX)

 

Botulinum toxin may provide relief of spasticity without the systemic adverse effects of other antispasticity agents. This agent binds to receptor sites on the motor nerve terminals and, after uptake, inhibits the release of acetylcholine, blocking the transmission of impulses in neuromuscular tissue.

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Contributor Information and Disclosures
Author

Jasvinder Chawla, MD, MBA Chief of Neurology, Hines Veterans Affairs Hospital; Professor of Neurology, Loyola University Medical Center

Jasvinder Chawla, MD, MBA is a member of the following medical societies: American Academy of Neurology, American Association of Neuromuscular and Electrodiagnostic Medicine, American Clinical Neurophysiology Society, American Medical Association

Disclosure: Nothing to disclose.

Chief Editor

Selim R Benbadis, MD Professor, Director of Comprehensive Epilepsy Program, Departments of Neurology and Neurosurgery, Tampa General Hospital, University of South Florida College of Medicine

Selim R Benbadis, MD is a member of the following medical societies: American Academy of Neurology, American Medical Association, American Academy of Sleep Medicine, American Clinical Neurophysiology Society, American Epilepsy Society

Disclosure: Serve(d) as a director, officer, partner, employee, advisor, consultant or trustee for: Cyberonics; Eisai; Lundbeck; Sunovion; UCB; Upsher-Smith<br/>Serve(d) as a speaker or a member of a speakers bureau for: Cyberonics; Eisai; Glaxo Smith Kline; Lundbeck; Sunovion; UCB<br/>Received research grant from: Cyberonics; Lundbeck; Sepracor; Sunovion; UCB; Upsher-Smith.

Acknowledgements

Nestor Galvez-Jimenez, MD, MSc, MHA Chairman, Department of Neurology, Program Director, Movement Disorders, Department of Neurology, Division of Medicine, Cleveland Clinic Florida

Nestor Galvez-Jimenez, MD, MSc, MHA is a member of the following medical societies: American Academy of Neurology, American College of Physicians, and Movement Disorders Society

Disclosure: Nothing to disclose.

Stephen T Gancher, MD Adjunct Associate Professor, Department of Neurology, Oregon Health Sciences University

Stephen T Gancher, MD is a member of the following medical societies: American Academy of Neurology, American Neurological Association, and Movement Disorders Society

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Medscape Salary Employment

Acknowledgments

The author is extremely grateful to patients with Pelizaeus-Merzbacher disease and their families for their help and support of Pelizaeus-Merzbacher disease research and to the Pelizaeus-Merzbacher Disease Foundation, the National Institutes of Health, and the Children's Research Center of Michigan for financial support.

References
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  15. Mori T, Mori K, Ito H, Goji A, Miyazaki M, Harada M, et al. Age-related changes in a patient with Pelizaeus-Merzbacher disease determined by repeated 1H-magnetic resonance spectroscopy. J Child Neurol. 2014 Feb. 29(2):283-8. [Medline].

 
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T2-weighted magnetic resonance imaging (MRI) scan of a child aged 10 months with duplication of the proteolipid protein (PLP) gene; note the high-intensity signal throughout the cerebral white matter.
T2-weighted magnetic resonance imaging (MRI) scan of a man aged 41 years with duplication of the proteolipid protein (PLP) gene; note the increased white matter signal, as well as diffuse atrophy.
T2-weighted magnetic resonance imaging (MRI) scan of a man aged 20 years with connatal Pelizaeus-Merzbacher disease due to a Pro14Leu mutation; note the severe reduction in white matter volume, as well as the increased white matter signal.
T2-weighted magnetic resonance imaging (MRI) scan of a boy aged 17 years with null mutation of the proteolipid protein (PLP) gene; note the more subtle increase in signal intensity relative to that seen in the previous images, and observe that the volume of white matter is normal.
 
 
 
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