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China’s 1st Rare Disease Registry

January 21, 2013

There are currently no existing registries in some countries. China is an example of a country with no existing registry up until recently. In the August 2011 issue of The Lancet, there is correspondence that discusses China’s 1st rare disease registry. The purpose of China’s 1st web-based registry is to connect patients and doctors, to provide information, and to gather data on patients. The story is fascinating.

The development of China’s 1st rare disease registry starts in May 2010, by a patients’ advocacy group for patients with Osteogenesis Imperfecta (OI). The name of the advocacy group is China-Dolls Care and Support Association. OI is a rare disease where patients have what is referred to as “brittle bone disease”. The bones of patients with OI get fractured easily due to the genetic bone disorder which strikes about 1 in 10,000 – 15,000 people. An easy bump can make OI patients’ bones break.

The origin of the name of the group, “china dolls” is interesting. ”China dolls” or “ciwawa” is what the group of people who has OI is known as, because “for them a fall, a collision with a hard object, or even a severe sneeze can end in broken bones (at bottom of webpage) .”

According to the FDA Orphan Products Designation Database, the following are drugs that have orphan drug designation for OI but not FDA approval :

Row #

Generic Name

Designation Date

Indication

1

     alendronate 03-31-2003 Treatment of osteogenesis imperfecta in   pediatric patients 4 years of age and older

2

     risedronate sodium 12-18-2006 Treatment of patients with osteogenesis   imperfecta.

References

Handle With Care By Jodi Picoult

Osteogenesis Imperfecta Awareness Week, May 4 – 11, 2013

Osteogenesis Imperfecta (OI) Foundation

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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