[ad_1]
In this photo from February 12, 2019, Meghan Waldron is walking down the street in Boston. Waldron is a student at Emerson College and suffers from progeria, one of the rarest diseases in the world. The first treatment was approved for progeria on Friday, November 20, 2020. The United States Food and Drug Administration on Friday approved Zokinvy, which tests show prolongs patients’ lives by an average of 2.5 years. (Suzanne Kreiter / The Boston Globe via AP)
TO Newsroom
UPDATE 9:19 am PT – Sunday, November 22, 2020
A breakthrough drug for the ultra rare genetic disease known as progeria has been approved by the Food and Drug Administration (FDA). The oral capsule, which goes by the name Zokinvy, received the green light on Friday.
Recent news! First treatment for a rare, rapidly aging disease #Progeria receives FDA approval! https://t.co/At4DdNUyAO pic.twitter.com/Kyr1zD1G9X
– The Progeria Research Foundation (@Progeria) November 20, 2020
The FDA has said it should reduce the risk of death in people with a fatal diagnosis. On average, it increases life expectancy by two and a half years. The research behind the treatment was largely funded by the Progeria Research Foundation (PRF).
“Hopefully the time will come when we will talk about progeria as something in the historical books that we understand,” said Dr. Francis Collins, director of the National Institutes of Health. “Not just how to treat it to make it a little better, but a cure and that’s a big task.”
With this latest development, PRF has moved closer to that goal. Once the drug becomes widely available, the approximately 400 people living with the disease worldwide, including 20 in the United States, will be able to breathe a little easier knowing that scientists are on the verge of a cure.
Progeria, also known as Hutchinson-Gilford Progeria syndrome, has been described by the FDA as a “rare genetic disease that causes premature aging, death and has a debilitating effect on people’s lives.”
At the molecular level, it is caused by a buildup of defective progerin or a progerin-like protein in cells. It causes vomiting, nausea, abdominal pain, fatigue, strokes, heart problems and more.
On average, children with progeria die around the age of 14. Fortunately, we hope that will change as Zokinvy hits the market after conducting four meticulous global studies over a period of more than 10 years.
MORE NEWS: Texas Prepares To Distribute Pfizer Vaccine Once Available
[ad_2]
Source link