Orphan disease is a global medical as well as social problem. Patients and their family members are often isolated geographically and mentally. Practicing physicians as well as researchers are also isolated and their clinical and research practice are difficult because of the rarity and limited information of the disease. Neutral Lipid Storage Disease (NLSD) and Triglyceride Deposit Cardiomyovasculopathy (TGCV) is such a disease. In the First International Symposium on NLSD/TGCV, held on November 26, 2011 in Kyoto, Japan, we had a consensus that we need to construct the international co-operative group to overcome this intractable diseases.
Here, I am pleased to inform you that we will have the Second International Symposium on April 19-20, 2013, in Osaka, Japan. At the second one, we would like to move forward to further understand their pathophysiology and consider possible therapeutic strategies. We will exchange progress we recently made. Furthermore, I would like to discuss with colleagues about the international registry system, which is essential to know the natural history of and test the effect of any therapeutic strategies on these rare diseases.
Finally, “rare disease” is not just an orphan. It is very often that the knowledge from rare diseases can be applied to commonly-observed diseases to understand their pathophysiology. TG deposition in diabetes mellitus, which is pandemic, will be one of the focuses to be discussed.
The Osaka University Nakanoshima Center is located in the center of downtown in Osaka and close to the Osaka Castle. I hope that you can enjoy spring blossoms and cultures in Osaka.
I am looking forward to seeing you soon.
Ken-ichi Hirano, MD, PhD
Department of Cardiovascular Medicine
Osaka University, Osaka, Japan
The 2nd International Symposium on Triglyceride Deposit Cardiomyovasculopathy
& Neutral Lipid Strage Disease
19-20th Apr. 2013
at Osaka University Nakanoshima Center