While the development of orphan drugs for rare diseases has skyrocketed in the last 30 years, Dr. Scott Gottlieb, Commissioner of the Food and Drug Administration is recently questioning the…
Originally reported by KVUE ABC, the race is on for the Rosenberg family to raise the funds that will benefit their 3-year-old child suffering from a rare brain disease known…
It was a Christmas miracle for the Garcia family as their infant quickly recovered and was discharged from the hospital to enjoy the holiday at home, reported DailyMail. 1-year-old baby…
High school student, Molly Alter, fell limp while braiding her best friend, Jackie Acierno’s hair, and it was the heroic actions of Jackie to perform CPR that saved her life,…
Happy holidays to the Vallecalle family, who are rejoicing in the fact their baby girl is cured of the rare disease, hyperinsulinism. Not only that, but they finally get to…
An amazing 6-year-old boy from Bradford, who was diagnosed with a rare heart condition called LEOPARD Syndrome, creatively started a fund raising campaign revolved around Christmas and singing to raise…
Reported by The Washington Post, six years ago Randall Jordan-Aparo was found dead after being sprayed down with a debilitating chemical when wrongfully placed into an isolation cell. According to…
According to The Washington Post, on Tuesday the Food and Drug Administration, FDA, made history by approving a gene therapy for childhood blindness, which will change the lives of many…
Jay and Kathy Keller of Franklin, Idaho are getting creative this year with their donation efforts, reports WENY News. The Kellers are asking for people to write Christmas letters to…
Originally reported by Fox 17 Nashville, many Tennessee residents stress that a new tax reform bill will eliminate continued drug advancement and development for many rare diseases. The House and…
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