First-ever Treatment for Progeria Discovered!!

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“PRF is a good example of an organization successfully enabling translational research, moving from gene discovery to clinical treatment at an unprecedented pace,” said Dr. Kieran. “From 1999, when the organization was founded, to today, PRF has identified the genetic mutation that causes the disease, funded preclinical research, completed this trial, initiated a second trial, and is currently working with our team at Boston Children’s Hospital to plan yet another trial with drugs that, like FTIs, have shown exciting results in Progeria cells and animal models. That’s an awesome track record of accomplishment.”

http://www.progeriaresearch.org/first-ever-progeria-treatment.html