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#RareDisease close-up: Tuberous Sclerosis

In Whitepapers by Caroline HornbyLeave a Comment

 

 

 

 

tuberous sclerosis alliance rare disease whitepaper Tuberous Sclerosis is a genetic disorder that causes tumors to form in many different organs, primarily in the brain, eyes, heart, kidney, skin and lungs. The disease affects some people severely, while others are so mildly affected that it often goes undiagnosed. The Tuberous Sclerosis Alliance provided this whitepaper on ‘Unlocking a cure for Tuberous Sclerosis complex: An assessment of scientific progress and research needs.’

Download the whitepaper here.

If you want to learn more about:

  • Methodology
  • Defining a “cure” for TSC
  • Areas of TSC research with significant progress
  • Areas in need of additional research
  • Unmet medical needs of individuals with TSC
  • Barriers to progress in TSC research

…then you need to download the whitepaper now.

The Tuberous Sclerosis Alliance concludes that “research on TSC is poised for providing new therapies that modify the progression of the disease, and possibly even prevent some of the most devastating manifestations. It will likely not be possible to unlock a cure for TSC in the near future, but with focused research efforts new treatments will significantly improve the lives of those affected, and will provide clues for treatment of other disorders.”

The Tuberous Sclerosis Alliance will be attending the World Orphan Drug Congress USA this April. Register now to meet with them and other leading rare disease patient groups.

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