According to a story from news-medical.net, a recent study that the reduced expression of miR-125a-5p results in an increase in the concentration of the protein TIMP-1. Prior research has associated…
February is Eisenmenger Syndrome Awareness month. Eisenmenger syndrome is very rare and devastating, so it deserves our attention. Let's get to know a little more about this heart condition. Eisenmenger syndrome…
February 16, 2018 Governor Herbert Declarations: Rare Disease Day on February 28th Undiagnosed Rare Disease Day on April 29th Park City, Utah. (February 16, 2018) – Rare and Undiagnosed Network…
Dangerous forms of brain cancer such as glioblastoma are almost impossible to treat. However, new research by Dr. Roel Verhaak could change that. Glioblastoma is the most aggressive form for…
Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
According to an article from news-medical.net, researchers are beginning to delve into the causes of a rare lung disease called primary ciliary dyskinesia. This research is revealing previously unknown information…
The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…
Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
Sophia Campa-Peters, a little 9-year-old suffering from a rare disease that resulted in several strokes, had a successful brain surgery just a few weeks ago, as reported Lubbock Online. The…
University of Glasgow scientists might have just come across an incredible new approach to providing patients with pharmaceutical medications on demand, reported first by International Business Times. This new technology…
Zaida was 3-years-old attending her normal yearly check-up with expectation that all was well, yet they quickly learned six days later of her terrifying cancer diagnosis, reports Daily Camera. Little…
Easton was born at thirty-six weeks gestation on January 6, 2017. He was almost immediately taken to the NICU for something called PPHN (Persistent Pulmomary Hypertension), Easton was intubated for…
February 14th is Valentine's Day! Make sure to treat your valentine to a special day - and while your at it, spread the word that February 14th is also National…
According to a story from Newswise, The University of Adelaide in Australia received a $1.19 million grant in order to spearhead an international research project. The project's aim is to…
There are fifty genetic diseases that can be characterized as lysosomal storage diseases (LSDs). Gaucher disease can be characterized as such. LSDs make up about one in every 8,000 births…
On January 30th, pop singer Jordin Sparks' step-sister Bryanna Jackson-Frias lost her battle with sickle cell anemia, reported People. Complications surfaced that sent her to the ICU. Jordin posted to…
My name is Amy, and I’m a 37 year old single mom of two beautiful girls, ages 8 and 13. I work full time as a business analyst for a…
A new effort to raise awareness and funding for rare disease is underway. SMA News Today reported that NORD, the National Organization For Rare Disorders, organized this campaign to challenge…
In an episode CNN's webseries LoveStory, titled Love Accessibly - we follow Kristen as she looks for love in New York City as a woman in a wheelchair. With her…
Still getting over that crazy Super Bowl game?! If you're an Eagles or Patriots fan, you might need abit more time to gloat/recover - but for the rest of us,…
Welcome back, Patient Worthians! This week, we have an opportunity for Parkinson's patients, and an update on an Amyloidosis app. We also have two stories of teenagers whose lives suddenly…
"Ok, well first of all...I know nothing. But if your kids are anything like me, they're going to break a lot of toilets!" Those are the starting words writer and…
Emma and Tripp Mattingly share the same infectious personalities of energy and happiness, but they also share the same rare genetic disease known as LPIN1, reports The Lebanon Enterprise. Living…
The Patients as Partners EU Conference, put on by The Conference Forum, took place at the end of last month in London. A diverse group met to collaborate and discuss…
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