Jump to content

Talk:Glycogen storage disease type II

Page contents not supported in other languages.
From Wikipedia, the free encyclopedia

Some text in this article was originally taken from http://www.ninds.nih.gov/disorders/pompe/pompe.htm (public domain)

note

[edit]

Can we add carrier frequency? Preferably in the first paragraph. neffk 19:10, 10 January 2007 (UTC)[reply]

Source for results of LOTS?

[edit]

Is there a source for these results? Peer-reviewed, preferabbly...--134.102.20.212 (talk) 08:24, 5 October 2009 (UTC)[reply]

Prognosis section

[edit]

This section contains only one source, but reads as if two studies were done. The first (Wagner, 2007) is summarised in the second paragraph. The second, which is not obviously linked to the first (though they may be the same study), is then discussed in paragraphs 3, 4 and 5, but without any citation. I suspect the latter is the same as the former, but can't tell. If they are, the text needs simplifying so that repetition is avoided. If they are not (as suspected by the anon above), the second study needs proper citation.

On a related point, the unsourced 2007 study is described as reporting "initial results". Given that it was publishing in 2007, presumably some time after the results were originally collected, are there "later results" that could now be added in? I ask in small part because there's apparently a (Harrison Ford) film coming out this week in which "curing" the disease is the focus. Cheers, --PLUMBAGO 09:57, 20 January 2010 (UTC)[reply]

S/S for late onset Pompe

[edit]

Should Hair Loss be added for the signs and symptoms? --Ely23 (talk) 09:56, 10 April 2011 (UTC)[reply]

[edit]

Hello fellow Wikipedians,

I have just modified one external link on Glycogen storage disease type II. Please take a moment to review my edit. If you have any questions, or need the bot to ignore the links, or the page altogether, please visit this simple FaQ for additional information. I made the following changes:

When you have finished reviewing my changes, you may follow the instructions on the template below to fix any issues with the URLs.

This message was posted before February 2018. After February 2018, "External links modified" talk page sections are no longer generated or monitored by InternetArchiveBot. No special action is required regarding these talk page notices, other than regular verification using the archive tool instructions below. Editors have permission to delete these "External links modified" talk page sections if they want to de-clutter talk pages, but see the RfC before doing mass systematic removals. This message is updated dynamically through the template {{source check}} (last update: 5 June 2024).

  • If you have discovered URLs which were erroneously considered dead by the bot, you can report them with this tool.
  • If you found an error with any archives or the URLs themselves, you can fix them with this tool.

Cheers.—InternetArchiveBot (Report bug) 03:45, 20 October 2017 (UTC)[reply]

[edit]

Hello fellow Wikipedians,

I have just modified one external link on Glycogen storage disease type II. Please take a moment to review my edit. If you have any questions, or need the bot to ignore the links, or the page altogether, please visit this simple FaQ for additional information. I made the following changes:

When you have finished reviewing my changes, you may follow the instructions on the template below to fix any issues with the URLs.

This message was posted before February 2018. After February 2018, "External links modified" talk page sections are no longer generated or monitored by InternetArchiveBot. No special action is required regarding these talk page notices, other than regular verification using the archive tool instructions below. Editors have permission to delete these "External links modified" talk page sections if they want to de-clutter talk pages, but see the RfC before doing mass systematic removals. This message is updated dynamically through the template {{source check}} (last update: 5 June 2024).

  • If you have discovered URLs which were erroneously considered dead by the bot, you can report them with this tool.
  • If you found an error with any archives or the URLs themselves, you can fix them with this tool.

Cheers.—InternetArchiveBot (Report bug) 16:38, 20 December 2017 (UTC)[reply]

citations needed in treatment section

[edit]

I think this article would benefit from inclusion of more precise information on the efficacy of Myozyme, and clear citations of the underlying data. The reference 9 - authored by Wagner is NOT a study, it is merely an editorial discussing a trial. This is a poor citation. The underlying trials would be better references.


The FDA approval of Myozyme in April 2006 is at: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2006/125141s000_MyozymeTOC.cfm In the FDA approval, under 'printed labelling', the efficacy was supported by discussion of 2 pivotal trials. The first trial discussed is one including 18 patients, and appears to be this study with first author Kishnani: https://n.neurology.org/content/68/2/99/ (It appears this study was completed prior to FDA approval and results known and used in the FDA approval, but not publicly published until after FDA approval) This is not a placebo controlled randomized trial, but rather against matched historical controls.

The second study referenced in the FDA printed labelling was described as an ongoing multicenter trial that enrolled 21 patients. I believe the study the FDA described was eventually publicly published as this study, with the described characteristics (for example: 16 free of invasive ventilation at baseline): https://www.nature.com/articles/gim200930


I think these 3 primary source materials would be important to include in this article, and would probably suffice for the 'citation needed' in the third paragraph of the section 'Treatment'

FDA approval Myozyme 2006 

https://www.accessdata.fda.gov/drugsatfda_docs/nda/2006/125141s000_MyozymeTOC.cfm

Kishnani 2007 

https://n.neurology.org/content/68/2/99/

Nicolino 2009 

https://www.nature.com/articles/gim200930


Parenthetically, I have qualms about the terminology "Myozyme treatment clearly prolongs ventilator-free survival and overall survival", since in precise medical terminology the word 'clearly' connotes a statistically inference generally based on placebo controlled trials. The two studies referenced were NOT placebo controlled, but against matched historical comparators. — Preceding unsigned comment added by 70.20.25.26 (talk) 14:49, 30 April 2020 (UTC)[reply]

[edit]

The citation [1] to medlineplus.gov should lead to https://medlineplus.gov/genetics/condition/pompe-disease/ but instead leads to https://medlineplus.gov/genetics/condition=pompedisease/ where it shows an error page 108.30.153.109 (talk) 01:20, 30 October 2023 (UTC)[reply]

Retrieved from "https://en.wikipedia.org/w/index.php?title=Talk:Glycogen_storage_disease_type_II&oldid=1202325530"