RE: Current Therapy for Charcot-Marie-Tooth Disease

[Guest guest]

Where can I get an entire reading of this abstract?

Mandy

Current Therapy for Charcot-Marie-Tooth Disease

Abstract from Curr Treat Options Neurol. 2005 Jan;7(1):23-31.

Current Therapy for Charcot-Marie-Tooth Disease. (mentions Vitamin C and

onapristone)

Grandis M, Shy ME.

Department of Neurology, Wayne State University, 4201 Saint Antoine Street,

Detroit, MI 48201, USA.

Charcot-Marie-Tooth (CMT), or heritable peripheral neuropathies, is among the

most frequent genetic neuromuscular disorders, with a prevalence of

approximately 1:2500. Since 1991, remarkable advances have occurred in

determining the precise genetic cause of many forms of CMT and in generating

animal models of many of these disorders. However, these advances have not yet

resulted in cures for CMT.

Recently, potential treatments for the most common form of CMT, CMT-1A, have

been shown in rodent models of the disorder. Treatment with onapristone, a

progesterone antagonist, has improved the neuropathy of the CMT-1A rat.

Treatment with large doses of ascorbic acid (vitamin C) has improved the

neuropathy of the CMT-1A mouse. Multicentric trials with ascorbic acid are

likely to start in the near future to assess if vitamin C supplementation is

effective and what is the dosage required in humans to improve neurologic

disability. Because of potential side effects with antiprogesterone therapy,

particularly in women of child-bearing age, research is actively proceeding with

progesterone antagonists to develop safe medications that also can be used in

clinical trials of CMT-1A.

Although no cures are available for CMT, there are many important treatments

available for patients with CMT that can improve their quality of life and help

them maintain their independence. Some of these therapies involve physiatry and

orthopedic surgery. Others involve pain management. Lastly, there are potential

concerns about medications or lifestyle issues that may exacerbate CMT. All of

these issues will be discussed.

[Guest guest]

Mandy,

Take this link to the journal - Current Treatment Options in Neurology 2005,

7:23-31 You have some choices. Instant access for $25 or perhaps you can

contact the authors and wait for them to send you a pdf version - if they have

them yet. Seeing this is actually a January publication, they might not. Plus

with the holidays they might be on break. Another way to go is to ask your

librarian to get it for you - may take several days/may take less, depending on

your library/hours/staff, holidays, etc.

http://www.treatment-options.com/cto_linkout_frmst.cfm?issn=1092-8480 & vol=7 & page\

=23

Gretchen

[Guest guest]

Here is the link to the article. What was posted is all I could find about

this article.

http://www.treatment-options.com/cto_linkout_frmst.cfm?issn=1092-8480

<http://www.treatment-options.com/cto_linkout_frmst.cfm?issn=1092-8480 & vol=7

& page=23> & vol=7 & page=23

Jay

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Re: Current Therapy for Charcot-Marie-Tooth Disease

Where can I get an entire reading of this abstract?

Mandy

Current Therapy for Charcot-Marie-Tooth Disease

Abstract from Curr Treat Options Neurol. 2005 Jan;7(1):23-31.

Current Therapy for Charcot-Marie-Tooth Disease. (mentions Vitamin C and

onapristone)

Grandis M, Shy ME.

Department of Neurology, Wayne State University, 4201 Saint Antoine

Street, Detroit, MI 48201, USA.

Charcot-Marie-Tooth (CMT), or heritable peripheral neuropathies, is among

the most frequent genetic neuromuscular disorders, with a prevalence of

approximately 1:2500. Since 1991, remarkable advances have occurred in

determining the precise genetic cause of many forms of CMT and in generating

animal models of many of these disorders. However, these advances have not

yet resulted in cures for CMT.

Recently, potential treatments for the most common form of CMT, CMT-1A,

have been shown in rodent models of the disorder. Treatment with

onapristone, a progesterone antagonist, has improved the neuropathy of the

CMT-1A rat. Treatment with large doses of ascorbic acid (vitamin C) has

improved the neuropathy of the CMT-1A mouse. Multicentric trials with

ascorbic acid are likely to start in the near future to assess if vitamin C

supplementation is effective and what is the dosage required in humans to

improve neurologic disability. Because of potential side effects with

antiprogesterone therapy, particularly in women of child-bearing age,

research is actively proceeding with progesterone antagonists to develop

safe medications that also can be used in clinical trials of CMT-1A.

Although no cures are available for CMT, there are many important

treatments available for patients with CMT that can improve their quality of

life and help them maintain their independence. Some of these therapies

involve physiatry and orthopedic surgery. Others involve pain management.

Lastly, there are potential concerns about medications or lifestyle issues

that may exacerbate CMT. All of these issues will be discussed.