It's been 35-years since the passing of the Orphan Drug Act, and creation of the National Organization for Rare Disorders (NORD), reports PR Newswire. Even though these organizations recognize there…
Joy Littlesunday is just one of many Navajo newborns who were screened and thus saved from severe combined immunodeficiency by a successful bone marrow transplant at UC San Francisco, reported UCSF…
A new study suggests that the antidepressant medication clomipramine may function to relieve symptoms of multiple sclerosis. In this instance, the drug was tested to treat progressive multiple sclerosis, in…
Around 15,000 people are to receive free eye treatment thanks to the efforts of Senator Mustafa Bukar. The senator represents the Katsina North Senatorial Zone. Senator Bukar announced the event…
Maria Menounos's talent has graced the TV screen, American journalism and occasionally, the world of professional wrestling. With WWE, she serves as a hardcore ambassador to the company. She's a…
Happy New Years, Patient Worthians! New year, new you... new treatment news? This week, we have an article about a molecule that could change the way we treat cystic fibrosis.…
Pharmaceutical company, Santen, has been developing a drug, intravitreal sirolimus, to treat noninfectious posterior segment uveitis. Uveitis is a type of eye inflammation, in which the uvea, which is the middle part…
NORD, the National Organization for Rare Diseases, sent out their happiest wishes for the holidays, their utmost gratitude for 2017, as well as their plan of attack for the New…
After over a decade, Debbie Zelman, founder of Debbie's Dream Foundation, succumbed to her stomach cancer at age fifty. From the beginning, Debbie's prognosis was not good. When the cancer…
According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…
According to a story on PRNewswire, the biopharmaceutical company, Enzyvant, kicked off their Farber Disease Natural History Study in October. Acid ceramidase deficiency, also known as Farber disease, is an obscure…
When Avery was born, it didn't take long for her parents to realize that there was something different about her. She was not moving as much as most babies do,…
Just like any other 7-year-old little boy, Ethan Perkins has big dreams. He loves trucks and reading, and wants to study fossils as a paleontologist reported KXLY. He also wants to be…
Technology is hurting us. That's the claim made many times over the years. Smart phones may not have as many risks as some fear, but they can be a significant…
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…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
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…
Everyone with Facebook surely remembers when people were posting videos of themselves having ice cold water dumped on their heads. But, does everyone remember what it was for? The Ice…
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…
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…
The Washington Post published a bizarre story earlier this month that is equal parts interesting and terrifying! A US National Security Agency counterintelligence officer (AKA a spy) is claiming that while…
Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
A video of former US Vice President Joe Biden on the talk show The View comforting co-host Meghan McCain has been making the rounds on the Internet the last few…
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