Living with a rare disease is no easy task, as most of our readers may agree. Not only are rare diseases difficult to diagnose because of the expertise needed to spot them, but treatments can be incredibly expensive or even non-existent. This often puts a double-fold stress on people living with rare diseases because it could mean the difference between prolonging life and living the rest of your life with an untreatable disease.
To help bring hope though to people with rare diseases all over the world, not just here in the United States, Pfizer has announced its plans to expand research and development of therapies and treatments for patients with rare diseases such as neuromuscular and hematologic diseases.
By expanding research in the area of rare diseases, the pharmaceutical giant hopes to get a better understanding of how rare hereditary diseases are passed through generations. Once this key piece of information is understood, researchers believe that they can utilize gene therapy platforms to help treat a wider range of medical conditions in the years to come.
As we said above and as so many of our readers may know firsthand, there are a large number of rare diseases out there -- some of which we have highlighted already on this blog -- that can be incredibly debilitating or have a high mortality rate. While this may be disconcerting now, if Pfizer is successful in unlocking gene therapy treatments that could be applied to other diseases, then it's possible that a new wave of hope could be just over the horizon.
Source: Market Watch, "Pfizer Expands Rare Disease Research with Establishment of Gene Therapy Platform," Dec, 8, 2014