Mom. “It is tremendously energizing,” said Dr. Leslie Gordon, the medical director and principal investigator of the Progeria Research Foundation. [Figure] Dr. Sam was diagnosed with progeria in 1998. Adding valuable information from Eiger enabled the team to prove to the FDA that when Zokinvy is administered to children with progeria, it does extend their lives. "We make a really great team," says Audrey Gordon, who is Sam's aunt and the foundation's president. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML. “That’s an 88 percent decrease in the risk of death during the time the kids were being treated,” says Dr. Kleinman. Meghan’s storyOne of those long-time lonafarnib trial participants is Meghan Waldron, now a 19-year-old sophomore in college studying creative writing. Children with Progeria die of heart disease at an average age of thirteen years. Boston Children’s is now running the largest progeria drug trial in history. His mother, Dr. Leslie Gordon, is researching new treatments for the condition. Sam was diagnosed with Hutchinson-Gilford progeria syndrome (progeria) when he was twenty-two months old. Now, by creating a login below, you can customize your homepage to the rare conditions that are most important to you. Sam’s father, Dr. Scott Gordon, is chairman of the board. Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare, fatal, segmental premature aging syndrome caused by a mutation in LMNA that produces the farnesylated aberrant lamin A protein, progerin. Thank you for agreeing to speak with me. Leslie Gordon is a co-founder of The Progeria Research Foundation and serves as the organization’s volunteer Medical Director. Although the first encouraging results of Zokinvy’s efficacy were published in 2012, there was no clear route to FDA approval. After becoming the foundation’s medical director, Gordon helped lead the team that discovered the gene mutation that causes progeria. Pediatrics. The late Sam Berns (centre) with his parents Scott Berns and Leslie Gordon at the 18th Annual Nantucket Film Festival in June 2013. Leslie Gordon is a co-founder of The Progeria Research Foundation and serves as the organization’s volunteer Medical Director. Accepted for Publication: March 6, 2018. Gordon and her husband, pediatrician Scott Berns, founded the Progeria Research Foundation. Leslie Smoot Importance Hutchinson-Gilford progeria syndrome (HGPS) is an ultrarare disorder associated with premature death due to cardiovascular events … LESLIE GORDON… When her toddler son Sam was diagnosed in 1998 with Hutchinson-Gilford progeria syndrome, she collaborated with noted … Most eventually succumb to heart disease and do not survive past their teens. According to Leslie Gordon, the co-founder and medical director at PRF, children with progeria live to … And now, Gordon … “We do not know if it is longer than that because some of the children who have been taking it since 2007 are still living,” says Dr. Kleinman. His mother, Dr. Leslie Gordon, is researching new treatments for the condition. In late November, the U.S. Food and Drug Administration (FDA) approved a repurposed cancer drug called lonafarnib to treat Hutchinson-Gilford progeria syndrome, better known as progeria — an ultra-rare incurable genetic disease associated with rapid aging. It's not a cure," says Dr. Leslie Gordon, an assistant professor of pediatrics at Brown University and medical director of The Progeria Research Foundation. "Leslie [is] in charge of the science aspect, I'm in charge of the fundraising and her husband, Scott, is the chairman of the board." For both the patients — and the researchers involved in the drug’s development and ultimate success — the story could not be more personal. The drug was initially developed by the Progeria Research Foundation, a non-profit organization founded by Drs Leslie Gordon and Scott Berns, the parents of Sam Berns. He spoke to NPR and various other media about progeria. … Grandson of Alice and Lewis Berns of CT, a A moving look at a couple's inspiring efforts to save their only son Sam from the rare and fatal premature-aging disease of progeria. We’ll find more and better treatments,” said Dr. Leslie Gordon, the foundation’s medical director. There are so many places I want to go.”. Patients born with progeria typically live to an age of mid-teens to early twenties. He was diagnosed at just 22 months old.