Clinical Trial of SMA Launches in Taiwan

Clinical Trial of Spinal Muscular Atrophy Launches in Taiwan

Spinal Muscular Atrophy (SMA), a genetic inherited disorder, suffers from motor  neuron regressed in the spine. One of the obvious symptoms is progressive muscular  weakness. It affects 1 in 10,000 live births, approximately 25 newborns affected by  SMA every year in Taiwan. 

There are three main types of SMA, classified according to severity and age of onset.  Type I SMA is known as the most severe type, beginning within the first 6 month of life.  Type II SMA, intermediate type, has its onset in 18 months of life. Type III SMA,  Kugelberg-Welander Disease, is relatively mild type which presents various symptoms,  from wheelchair-dependent patients to fully independent ones. 

It has been considered as no effective treatment to prevent muscular regression. However,  the ongoing clinical trial is launched in Taiwan which may lighten the dark on March 2007.  Kaohsiung Medical University Hospital administrates this clinical trial, using Hydroxyurea  in Spinal Muscular Atrophy type II and III, which is approved by National Department of  Health. Sixty patients affected by SMA type II and III will be enrolled for this clinical trial  which is 24-month, randomized, double-blind and placebo-controlled. 

TFRD is happy to see more and more clinical trials going on in Taiwan, helping more  patients and bringing hope to them. In the future, TFRD will help bulletin information of  rare diseases clinical trials that have passed the IRB approval. We look forward to more  and more treatment for patients.