Monday, January 11, 2016

Treating Achondroplasia: fourth year online

In this January, the blog is reaching four years since the first article. A lot of events has passed during this time. We have seen the first potential medication to treat achondroplasia entering clinical trials and several others being described in the literature that may bring relief to the many clinical complications seen in this and in other skeletal dysplasias.

The goal of the blog remains the same: to provide information about achondroplasia and the research for therapies for this skeletal dysplasia in a way that the hard language of the science is translated to a more accessible text. I know sometimes the articles here are still a bit hard to digest completely, but be sure I am always working to make them more understandable for the non-scientific reader.

Many of the articles come with an introduction that revisit the basics of achondroplasia. If the one you are reading, particularly the most recent ones, seems to be difficult to understand, I invite you to stop reading it and visit older articles in the index page. Let's give two examples. As I just wrote above, practically all articles come with references to the growth plate and FGFR3, and frequently the introduction makes a summary of the basics just to put the subject of that article in context. If you are having hard time understanding the growth plate and its relation with achondroplasia, why don't you try reading the first article published about it? It's here

Many people are interested in vosoritide and its clinical trials and this CNP-analogue has been the subject of several articles lately, as new information about its clinical development is published. However, some of the readers have trouble to understand the nature of this potential therapy for achondroplasia. If this is your case, please try to read one of the first articles about CNP published here in the blog.

The blog now has some new features. I have added two new pages to the page bar: Support & Information and Research. The first lists some of the main organizations dedicated to achondroplasia and/or rare diseases in the world. There, you may find relevant information for you or your community that may help you work on daily challenges or medical and/or social issues. The second lists the investigators working in fields that may bring new solutions for achondroplasia and other skeletal dysplasias in the future. Finally, the blog's home page now shows the three last articles published.

Thank you Maria Cristina Terceros for the strong help in translating the articles to Spanish. I also thank all the readers for the questions and highlights in their comments, as well as I am grateful for the discussions in our related Achondroplasia group in Facebook.  I am glad to see that the blog has reached 140,000 visits this month and I thank you visitors for the interest in the topics we review or discuss here. You are the reason of this blog. Happy new year!

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