Treatment
The cause of progeria (the genetic mutation in LMNA) was discovered only 9 years ago in 2003.
The treatment, an orally taken medication called Lonafurnib, is even newer.
Later in 2003, after the discovery of the mutation that causes progeria, the Progeria Research Foundation launched a study to observe the affects of the drug Lonafurnib, an orally taken medication made by Omerck & Co. originally produced for cancer treatment, on patients with progeria.
During this study, overseen by Mark Kieran, M.D., Ph.D., Director of Pediatric Medical Neuro-ology at the Dana Ruber/Children's Hospital Cancer Center, 28 of the 38 known children on the planet with progeria took Lonafurnib orally twice a day for two and a half years visited Boston every four months.
All of the patients showed significant improvement whether it be by weight gain (50% increase in annual weight gain), better hearing, stronger bones, or increased flexibility of blood vessels (increased by 35%).
The most important result out of these, is the increase in cardiovascular flexibility. This is because it decreases the effects of their cardiovascular disease (the leading cause of death among children with progeria) which decreases the death rate.
To view the study's results, click on the button below.
The treatment, an orally taken medication called Lonafurnib, is even newer.
Later in 2003, after the discovery of the mutation that causes progeria, the Progeria Research Foundation launched a study to observe the affects of the drug Lonafurnib, an orally taken medication made by Omerck & Co. originally produced for cancer treatment, on patients with progeria.
During this study, overseen by Mark Kieran, M.D., Ph.D., Director of Pediatric Medical Neuro-ology at the Dana Ruber/Children's Hospital Cancer Center, 28 of the 38 known children on the planet with progeria took Lonafurnib orally twice a day for two and a half years visited Boston every four months.
All of the patients showed significant improvement whether it be by weight gain (50% increase in annual weight gain), better hearing, stronger bones, or increased flexibility of blood vessels (increased by 35%).
The most important result out of these, is the increase in cardiovascular flexibility. This is because it decreases the effects of their cardiovascular disease (the leading cause of death among children with progeria) which decreases the death rate.
To view the study's results, click on the button below.
The discovery of Lonafurnib's new purpose has many optimistic about the future of a cure for progeria, but at this point, it is unknown how soon if ever a cure will be found.