Quick Search
  Home Journal Information Current Issue Past Issues Services Contact Us  
Articles
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy 
 
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy
  Qing Ke, Zheng-Yan Zhao, Jerry R. Mendell, Mei Baker, Veronica Wiley, Jennifer M. Kwon, Lindsay N. Alfano, Anne M. Connolly, Catherine Jay, Hanna Polari, Emma Ciafaloni, Ming Qi, Robert C. Griggs, Michele A. Gatheridge
 [Abstract] [Full Text] [PDF]   Pageviews: 4661 Times
 
Background: Advances in treatment for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) hold promise for children with these disorders. Accurate genetic diagnosis, early in the disease process, will allow these treatments to be most effective. Newborn screening (NBS) for SMA has been recommended in the United States, and a pilot DMD NBS program is underway in Hangzhou, China.
Data sources: A PubMed search, limited to the past 5 years, was conducted to identify: (1) therapeutic advancements for DMD/SMA approved by the United States Food and Drug Administration or the European Medicine Agency and (2) The status of NBS for DMD/SMA.
Results: We review the current state of approved treatments for DMD/SMA. We present recommendations regarding the future of NBS for these diseases, with a focus on the outcomes and challenges of SMA NBS in New York, USA, and the DMD NBS pilot program in Hangzhou, China.
Conclusions: Approved treatments for DMD and SMA may change the natural history of these diseases. Long-term studies of these treatments are underway. To avoid the known diagnostic delay associated with these disorders and provide optimal effectiveness of these treatments, early identification of patients through NBS will be necessary. Establishing comprehensive follow-up plans for positively identified patients will need to be in place for NBS programs to be successful.
 
  [Articles Comment]

  title Author The End Revert Time Revert / Count

  Username:
  Comment Title: 
 
   

 

     
 
     
World Journal of Pediatric Surgery

roger vivier bags 美女 美女

Home  |  Journal Information  |  Current Issue  |  Past Issues  |  Journal Information  |  Contact Us
Children's Hospital, Zhejiang University School of Medicine, China
Copyright 2007  www.wjpch.com  All Rights Reserved Designed by eb