It appears that rare diseases are getting more coverage in the media and bio-pharmaceutical companies are willing to take on the tasks of research and drug trials
.
http://www.phrma.org/working-together-we-can-produce-results
1. Ultragenyx, the company that is conducting the Sialic Acid (extended and immediate release) tablets for HIBM has started trading their stock on Wall Street now. They use the ticker symbol RARE. Here is a link on their presentation and an upcoming event. On March 5th they will be having a Health care conference of which it seems one could register for.
http://ir.ultragenyx.com/events.cfm
2. The LA Marathon will be happening on March 9th. and there is a site set up for those who would like to participate or donate.
http://www.crowdrise.com/runforhibm2014/fundraiser/sandradarvish
3. Ultragenyx is recruiting participants for a Combined Registry Prospective Natural History Study. They are recruiting participants in the United Kingdom, Canada, and the United States. I see also listed are France and Bulgaria however they are not recruiting yet. Here is the link:
http://clinicaltrials.gov/ct2/show/NCT01784679?term=hibm&rank=2
4. The National Institutes of Health still seems to be recruiting for their natural history study:
http://clinicaltrials.gov/ct2/show/NCT01417533?term=hibm&rank=4
5. There is support group on facebook for patients and family with HIBM. This is a very helpful group of people who offer tips on how to manage our disease. We also started a Google hangout group and this was quite informative to see and chat with others.
Please feel free to contact me if you need more information in this blog.
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