According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be…
According to a story by Mirror, the Rare Disease Centre at Birmingham Children’s Hospital is set to begin operations after a massive donation by readers of The Star Appeal. Readers…
According to a story from Beyond the Dish, a new experimental treatment for spinocerebellar ataxia was given the go-ahead from the U.S. Food and Drug Administration for testing. The investigational…
Who couldn’t use a little extra money now and again? Better still, what if earning that money was as easy as a mouth swab? According to a report on Journal…
According to a story from the FH Foundation, the non-profit organization has released a vital consensus statement regarding the diagnosis of familial hypercholesterolemia, which is the most prevalent genetic cause…
According to a story from cbc.ca, Cole Pringle is a 31-year-old man from Regina, Saskatchewan with spinal muscular atrophy, a debilitating illness that severely affects his mobility. He was diagnosed…
According to a story from Physician's Weekly, a recent study found that small amounts of invisible blood in stool increases the possibility of early death from other causes. The study…
According to a story from Science Daily, researchers have discovered a mechanism that drives the development of renal cell carcinoma. This mechanism could lead to new therapeutic approaches that take…
According to a story from Charcot-Marie-Tooth News, a recent study suggests that changes in the levels of two proteins in the body could serve as biomarkers to indicate the progression…
According to a public release from University of Texas Health Center at San Antonio on eurekalert.org, research using fruit fly tauopathy disease models has revealed a genetic "copy-paste" activity that…
For anyone who has had to step into a doctor's office, you know the litany of emotions that run through your body. Anxiety, confusion, worry, exasperation - and to say…
Ailani Banuelos is a 12-year-old with a dream. She not only wants to see the ocean but paddle in it. Living in Chicago made the dream fairly difficult. There simply…
Ashley Wibberly’s son Levi was born in September of 2013 and lived for just 20 months with Krabbe disease. Ashley couldn’t have seen it coming. Now, however, a new test…
What do Dave Winfield, Steve Garvey, and Don Baylor Jr. all have in common? Besides all being big names in major league baseball, each of the famous athletes is contributing…
Happy End-of-July! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributor, Tom Seaman. Next, we investigate the root…
Denise Albert is a busy woman. She founded The MOMs media company. She enjoys music, concerts, and time with her boyfriend. She is also a happily divorced mom of two…
An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to…
Pets brighten the lives of many people across the world. There are a variety of studies and pieces of evidence that point to pets improving the quality of life for…
According to a story from the Los Angeles Daily News, for children with debilitating rare illnesses like cerebral palsy, epilepsy, or muscular dystrophy, it can often take a long time…
According to a story from pm360online.com, the company PledPharma recently announced that its drug candidate Aladote has completed its first safety evaluation with a positive result. Aladote is in development…
Novartis has teamed up with Numinous Games to develop Galaxies of Hope, an app that is designed to support the neuroendocrine tumour (NET) community. You can read the original source…
Having a child was an exciting moment for the Blackwood family. Their son, Sam, is now nine years old. It was around his first birthday, however, that they started to…
Jaiden, a twelve-year-old boy, has been diagnosed with stiff skin syndrome, a rare disease that causes his skin to harden. His family has been sharing their experiences with the condition.…
According to a story from the Washington Post, a new technique for delivering genes into immune system T cells could make immunotherapy a much more accessible option for treating rare…
Happy Friday, everyone! We hope the weekend is bringing everyone some time to relax. Today, we're wrapping up the week with four stories we wanted to highlight this week. We…
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