According to a story from EPM Magazine, the pharmaceutical company Orchard Therapeutics received Rare Pediatric Disease Designation for its gene therapy drug candidate OTL-200. The therapy is in development for…
According to a story from MD Magazine, the FDA has recently approved the first multiple sclerosis treatment catered to children with the disease. In this instance, the agency did not…
Happy Friday, everybody! This week we're highlighting a celebration of rare moms, thoughts on the value of prayer in the rare community, and a PBC patient offering guidance on how…
According to a story from floridahospital.com, Huey Lewis, the front man and vocalist of the highly successful pop rock band Huey Lewis and the News, was recently diagnosed with Meniere's…
According to a story from azcentral.com, the Trump administration has decided to roll back some budget cuts that put the continued existence of regional Hansen's disease clinics in jeopardy around…
A woman from the UK, whose husband was diagnosed with Parkinson's disease many years ago, has the unique distinction of being able to detect the disease in patients through a…
According to a story from The Independent, the 60 years of blood donations from a single man may be directly responsible for saving the lives of 2.4 million babies. The…
Multiple sclerosis (MS) patients in the United States and other countries around the world are trekking to Russia to undergo a controversial treatment for the neurological disorder that some researchers…
This article contains spoilers The actor-turned-director John Krasinski’s new horror film A Quiet Place has received widespread acclaim, due in part to its representation of deafness. The film is set in post-apocalyptic…
Jordy Cernik isn't afraid of anything. It's not that he has some kind of super ability that makes him fearless or special secret agent training that defies normal reactions to…
The Vasculitis Foundation has released a video called “Connect the Dots” to raise awareness about vasculitis within the medical community, reports Newswise. Vasculitis is a frequently misdiagnosed or undiagnosed condition,…
According to a story from cafepharma.com, Akcea Therapeutics, Inc. recently announced that the FDA's Division of Metabolism and Endocrinology Products Advisory Committee voted to recommend approval of the therapy Waylivra…
According to a story from medicalxpress.com, a recent study from the University of Birmingham discovered that the progression of multiple sclerosis could be predicted by spinal fluid analysis. This could…
A year after being approved as the first new treatment for amyotrophic lateral sclerosis (ALS) in 22 years, Radicava is altering the way physicians treat the illness, which is also known…
The family of Tessa Jowell, a UK politician who recently passed away from a brain tumour, have commended the government for their commitment to improving diagnostic and treatment services for…
According to a story from the Hereford Times, a combination of misdiagnosis and a critical mistake to forgo a simple blood test led to the tragic death of Callum Cartlidge.…
Patients with juvenile idiopathic arthritis (JIA) who used a program to receive health education and help in taking the lead in their own disease management is winning praises for its…
In a press release, CURE Media Group announced the development of a 13-part series on carcinoid syndrome. A series of discussions involving both doctors and patients educating patients, caregivers and…
According to a story from Neuroscience News, the case of a woman with neck dystonia who was using a deep brain stimulation device called an implantable pulse generator (IPG) indicates…
According to a story from eurekalert.org, a recently study has helped reveal some of the genes that are associated with hemiplegic migraine headaches. About 15-20 percent of adults in the…
According to a story from the Leamington Observer, four-year-old George Young's first words were 'mama' and 'papa,' but he did not know how to say them for very long. George's…
Happy Pre-Mother's Day Everybody! This week we're spotlighting four articles from or about members of the rare community. We have a tribute to nurses from a rare mom recognizing their…
According to a story from Sickle Cell Anemia News, the St. Jude Children's Research Hospital just received $1 million in grant funding from The Links Foundation, which is one of…
According to a story from Newshub, Freda Evans learned early on that there were no treatments covered under the New Zealand health care system to treat her Pompe disease. Freda…
May is Lyme disease awareness month. Responding to an explosion in Lyme disease and tick-borne illnesses, the Tick-Borne Disease Working Group met for the first time in Washington, D.C. in December…
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