History has been made!
Parents with kids with a very rare disease created their own charity and that paid off!
Now, a clinical drug trial showed improvement in one or more areas of their condition. The frug, a farnesyl transferase inhibitor lonafarnib is first known treatment for children with Progeria.
The clinical trial , sponsored by the new foundation, showed that lonafarnib significantly improved the children’s’ rate of weight gain, bone structure, hearing and/or vascular stiffness. The vascular finding is the most exciting – improving the cardiovascular status of children with Progeria is critical, as heart attacks and strokes are the ultimate cause of death.