Friday, April 19, 2013

Orphazyme Announces Proposed Clinical Trial 
of rhHSP70 for NPC

~ Apr 19th, 2013 ~

Dear NPD Community, 
Orphazyme, a Danish biotech company, announced its intention to conduct a trial of rhHSP70 as a therapeutic intervention in NPC disease, at a scientific conference in Italy this week (15th-19th April 2013).  
Orphazyme has provided information for patients and families which will be generally available through patient organizations across the world. This information has been issued on the understanding that much has still to be confirmed / agreed by the regulatory authorities, so please be aware that some of the details may change. 
To assist you, we have produced an additional document that summarizes the main points. 
All documents can be found on the NNPDF website's Latest Research and Newsline pages. 
In order to facilitate communication further, Orphazyme intends to launch a web page for the trial and to include a “Frequently Asked Questions” section on the page.  As soon as this information is available, we will share it with you. Further information about Orphazyme can be found on their website:

Wednesday, April 3, 2013

Running for Rare Disease in Memory of Wylder Laffoon

Dear NPD Familes and Friends,

Running for Rare Diseases, an amateur running team comprised of Genzyme employees, is honoring our precious Wylder Laffoon (05/15/09 - 07/20/12, NPA) by o
ne of their members running in his memory at this years Boston Marathon!

Runner Phil Mederia chose to represent NPD and help to spread awareness, along with his team mates who are working with NORD to promote their project "Promote Early Diagnosis".

We encourage our families and friends to visit their Facebook and "Like" the page to show we stand with Phil and his team for their efforts to spread awareness for rare diseases and honor the memory of Wylder.

Please visit the Newsline page for links and further information.