New Podcast in Patient Perspectives Series: Searching for a Cure

LA JOLLA, Calif.--(BUSINESS WIRE)--Jul 10, 2009 - California Healthcare Institute:

                   
          WHO:

 

      Debra Miller, president and founder, Cure Duchenne
           
          WHAT:

 

      Patient Perspectives Podcast Series: Searching for a Cure
           
          WHEN:

 

      Friday, July 10, 2009
           
          WHERE:

 

      www.chi.org or Patient Perspectives Podcast Series

 

           
          DETAILS:

 

      CHI-California Healthcare Institute, The Burrill Report and the Children's Rare Disease Network released the latest episode of the Patient Perspectives Podcast Series: Searching for a Cure. Debra Miller, president and founder of Cure Duchenne, speaks about her son's journey through treatment after being diagnosed with Duchenne Muscular Dystrophy (DMD) and how his experiences inspired her husband, Paul, and her to start Cure Duchenne. DMD primarily affects young boys and causes progressive muscle loss; it is the leading genetic killer of children and most patients do not live past their teen years. As Miller and her husband explored treatment options and sought support, they found that existing patient advocacy organizations were often focused on palliative care and focused on academic research rather than working with industry to find a cure. In this episode of the Patient Perspectives Podcast Series, Miller speaks about new approaches to treating DMD and the problems of funding cutting-edge research in search of a cure.

 

                   
About Duchenne Muscular Dystrophy

 

  • One in every 3,500 boys worldwide (an estimated 20,000 babies annually) is afflicted with Duchenne Muscular Dystrophy.
  • Boys with Duchenne are typically diagnosed before the age of five. The disorder affects all ethnic and social groups. The gene mutation that causes Duchenne is usually passed from mother to son, but fully one-third of cases are spontaneous.
  • Patients with Duchenne experience muscle swelling and their joints become restricted. They are easily fatigued and have difficulty keeping up with others their age. Most boys with Duchenne need a wheelchair between the ages of 10 and 12.
  • The simplest of tasks, such as holding a fork or turning over in bed, becomes very difficult.
  • In the later stages of the disease, heart and breathing muscles begin to fail. Historically, most boys with Duchenne have not survived their teens.
  • There is no cure¦and it can happen in any family.
  • Gene therapy and stem cell research have made huge advances in just the last year. Researchers believe actual cures could be anywhere from five to 20 years away.
  • There is, however, a new class of drugs and therapies which will not cure the disease, but will fool the body into producing dystrophin or will upregulate other proteins that could take the place of dystrophin, while at the same time eliminate some of the damage that occurs in the muscle cells of Duchenne boys.

CHI's Patient Perspectives Podcast Series

CHI's Patient Perspectives Podcast Series examines patient experiences throughout the healthcare continuum, and touches on topics ranging from access to information and care, to breakthrough research improving quality of life, to solutions to the problems of rising healthcare costs and bureaucracy. CHI's podcasts increase awareness about the life-saving, world-class research being done in California.

About California Healthcare Institute

CHI represents more than 250 leading biotechnology, medical device, diagnostics, and pharmaceutical companies, and public and private academic biomedical research organizations. CHI's mission is to advance responsible public policies that foster medical innovation and promote scientific discovery. CHI's Web site is www.chi.org.

About The Burrill Report

The Burrill Report provides actionable market intelligence on the latest global developments and trends in the life sciences industry. Available bi-monthly, subscribers also benefit from monthly market updates and financial and business statistics provided in a special premium area on The Burrill Report Web site, www.burrillreport.com.

About the Children's Rare Disease Network

The Children's Rare Disease Network is an online community that provides children with rare diseases, their families, and the organizations that support them with the opportunity to communicate, collaborate, become educated and tap existing resources, to helping them with their daily challenges. Visit www.theprojectcharity.org.

 

Contact: CHI
Nicole Beckstrand, (858) 456-8881
beckstrand@chi.org
or
The Burrill Report
Peter Winter, (415) 200-8163
or
The Children's Rare Disease Network
Nicole Boice, (949) 305-8656
nicoleb@theprojectcharity.org

 

Posted: July 2009

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