Rare Disease patients are at the forefront of a genetic revolution in medicine.
As well as raising awareness, Rare Disease Day is a time to celebrate the achievements of those affected by such conditions. And now we have the heroism of rare disease patients and their doctors to thank for a treatment that rewrites faulty DNA.
As many of the rarest diseases in the world are caused by a single faulty gene, the early genetic pioneers figured this would be the easiest place to start their research. The fatal immune disorders known as SCID proved to be perfect candidates for gene therapy. In the past SCID was famous for confining David Vetter to a sterile bubble, in the future it will be heralded for launching a genetic revolution in medicine. With trials for numerous rare diseases now underway around the world and more still in development, such patients are trail-blazing a completely new medicine for mankind.
As the theme for this year's Rare Disease Day is research, I've made a video that gives an insight into the world’s first government funded gene therapy and what it means for those affected.
So I guess this edition is more of a Vlog than a Blog, please click on the photo to go to the VIDEO