Gene therapy has the potential to transform how we manage rare genetic diseases like hemophilia, Duchenne Muscular Dystrophy and others by addressing their root cause – genetic mutations.
The Power of Science behind gene therapy inspires our colleagues, like Bob Smith, SVP, Global Gene Therapy Business, to investigate this groundbreaking technology that could, if successful, bring disease-modification to millions of patients worldwide.
Did you know of 7,000 rare diseases, only 5% have treatments available?
On July 18th, Shire invited professionals local to Cambridge, MA to participate in Rare in Kendall Square: Collaborating to Advance Innovation in Rare Disease. Over 200 attendees gathered to network and weigh in on a panel discussion centered around the idea that partnerships can advance treatment options.
“Our fight against rare diseases cannot be waged alone – unique partnership models are critical,” said Flemming Ornskov, M.D., M.P.H. and CEO of Shire.
Guidelines Emphasize Respecting the Values and Independence of Patient Organizations
WASHINGTON, Sept. 28, 2012 / 3BL Media / PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) today issued a statement commending the Pharmaceutical Research and Manufacturers of America (PhRMA) for adopting principles to guide the interaction between pharmaceutical companies and patient organizations.