Living with a rare disease can be highly challenging,  particularly if it begins during childhood. However, rare diseases in children do not only harm the child but cause pain for entire families. Parents are forced to watch their children suffer without proper answers as to the cause of their suffering, as there is a lack of diagnosis and treatment despite scientific advances. In addition, rare diseases carry significant ethical burdens for the clinicians who treat the children, as they have to make the difficult decision as to whether or not to continue life support. 

Developing treatments for rare diseases in children takes work and we are delighted that Stemnovate has put this platform in place to accelerate research, develop treatments and partner with leading pharmaceutical companies to find new ways to treat these complex cases.

Please click the link to read more about their amazing work!