Hear Us Now: Rare Disease Families Making a Difference

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Hear Us Now: Rare Disease Families Making a Difference

Posted by Eileen O'Brien | 10:13 am on Wednesday September 12, 2012 | 6 Comments

The Hempels are one of the many rare disease families who are racing against time to save their children’s lives. Their twin daughters, Addison and Cassidy, have Niemann-Pick type C, a rare and fatal genetic disease that causes progressive neurological deterioration. A documentary about the family, Here. Us. Now., has been commissioned by the Ewing Marion Kauffman Foundation.

There is no treatment for Niemann-Pick type C, so the Hempels did their own research and discovered a simple compound, cyclodextrin, being used in mice. They petitioned the FDA to get permission to use it on their daughters. While not a cure, the Hempels feel that it has slowed the progression of the disease. In 2011, the couple started their own biotech company, Solution Therapeutics, and is seeking funding to start clinical trials of cyclodextrin. The Hempels’ story reminds me of those of several other empowered patients and families who have taken action and are making a difference.

LA screening
On Friday, September 28, 2012, the Global Genes Project will hold a free screening of the documentary at 7 p.m. at the Harmony Gold Preview House  in Los Angeles, California. A limited number of seats will be made available for this private screening.

About Eileen O'Brien

Eileen has more than 16 years of digital healthcare marketing experience. She is an opinion leader on social media and biopharma, and has been invited to speak at industry conferences and quoted in publications. She can be found on Twitter at @EileenOBrien and on Google+.

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