First China SMA Fair held in Beijing
( Chinadaily.com.cn )
The first China SMA Fair, hosted by the Meier Advocacy & Support Center for SMA and Beijing E-Town Biopharm Park, and supported by the Beijing Ruicy Gene Therapy Institute for Rare Diseases and the Science and Technology Bureau, was held in the Beijing Economic and Development area on August 6.
SMA, or spinal muscular atrophy, is a rare neuromuscular disorder characterized by loss of motor neurons and progressive muscle wasting. The disorder is caused by a genetic defect in the SMN1 gene, which encodes SMN, a protein widely expressed in all eukaryotic cells and necessary for survival of motor neurons. Lower levels of the protein result in loss of function of neuronal cells in the anterior horn of the spinal cord and subsequent system-wide muscle wasting. Spinal muscular atrophy manifests in various degrees of severity, which have in common progressive muscle wasting and mobility impairment. Proximal muscles and lung muscles are affected first. Other body systems may be affected as well, particularly in early-onset forms of the disorder. SMA is the most common genetic cause of infant death.
At the fair, the Beijing Ruicy Gene Therapy Institute for Rare Diseases used 3D printing to create an exoskeleton to address general concerns. The Institute, located in the Beijing E-Town Biopharm Park, is a non-profit research institute. It cooperates widely with overseas colleges, medical organizations and enterprises on difficult projects. It aims to use "molecular machine"-gene therapy and "intelligent machine"-wearable device to treat diseases.
Dr. John R. Bach and Dr. Kathryn J. Swoboda, two authorities in this area, attended the fair.
Dr. Bach specializes in treatment of neuromuscular and pulmonary diseases, and Dr. Swoboda concentrates on diagnosis and treatment of neuromuscular disorders, dyskinetic disorders and genetically inherited neurological diseases in childhood.
It was the first time the two professors have come to China and shared their knowledge of SMA.
An analytic report on Chinese SMA patients was released at the fair. The report notes that SMA always onsets before the age of two. Fewer than 7.5 percent of Chinese patients undergo pulmonary function tests and fewer 11.2 percent regularly revisit a hospital. In addition, only one child has received corrective scoliosis surgery. The treatment of Chinese SMA patients has a long way to go. As a good start, an "Angel dream plan" was promulgated at the fair: it will choose 60 dream achievements which are now highly difficult for SMA patients, and make them come true by diverse methods.
Many authoritative experts offered their knowledge about SMA at the two-day fair, and special education and exercise rehabilitation workshops were set up to spread knowledge of the disorder and help improve patients’ lives.
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