The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral…
I'm Celeste, and I'm sharing my story because too many ill people go undiagnosed and too many healthy people know nothing of it. Being chronically ill is like (for lack…
According to a story from Ovarian Cancer News, a recent study has allowed scientists to determine why some people appear to be resistant to poly ADP ribose polymerase (PARP) inhibitors.…
The not-for-profit healthcare organisation Sanford Health is planning to implement routine genetic testing for patients to identify a patient’s risk for a range of rare, and more common, diseases. The…
In the Victorian period, the British Royal family was known to carry haemophilia, leading to the condition also being known as ‘the royal disease’. Now, no members of the British…
Dogs make great pets and companions. They’re often loving and obedient. But could dogs also be the key to curing some forms of cancer? When many people think of dogs…
According to a story from news-medical.net, a recent study has revealed that an ingredient commonly found in toothpaste could help patients with cystic fibrosis. The study was conducted by researchers…
According to a story from PR Newswire, the pharmaceutical company BioMarin Pharmaceutical Inc. has recently announced that the U.S. Food and Drug Administration as issued approval for their product pegvaliase-pqpz, which…
According to a story from ktvq.com, Susan Henderson of Bozeman used to revel in painting vast, impressive canvases and teaching art classes geared towards children. Unfortunately, her diagnosis of chronic…
The Young family from Oregon, U.S. have used genetic screening to test their embryos for a variation of the BRCA gene that significantly increases the risk of breast cancer, reports…
My name is Jeremiah, and I have Postural Orthostatic Tachychardia (POTS). My body has been a source of significant challenges since I was very young. Asthma, unexplained rashes, passing out,…
According to a story from polygon.com, Microsoft has developed a new Xbox Adaptive Controller that is tailored towards players that have reduced use of their hands and fingers. While there…
The Melanoma Research Alliance (MRA) and The American Cancer Society (ACS) have announced the first recipients of their new research grants, reports PRNewswire. The grants have been set up as…
TGIF, everybody! This week we're sharing music videos by a singer who raises awareness for aHUS, a journey of a dysautonomia patient, and a story about a man who was…
According to a story from inhomelandsecurity.com, military veterans are roughly twice as likely to get amyotrophic lateral sclerosis (ALS), a lethal degenerative disease that kills in as little as two…
May has been designated Behçet's disease awareness month - but unlike so many of the rare diseases we cover, there are not any advocacy organizations dedicated to raising awareness and support…
A new treatment for cystic fibrosis has received a lot of media attention in the UK after the National Health Service (NHS) chose not to fund it due to its…
According to a story from the Washington Post, an outbreak of Nipah virus has claimed a total of ten lives so far in south India. Medical teams are starting to…
According to a story from Medical Xpress, the drug adalimumab (marketed as Humira), did not have a noticeable effect on aortic inflammation in patients with psoriatic arthritis or psoriasis. Humira…
According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…
According to a story from Cardiovascular Business, The FH Foundation, which is a frontrunning patient advocacy and research organization for familial hypercholesterolemia, recently announced that it has begun enrollment for…
Primary biliary cholangitis (PBC) is a rare disease, but you don’t have to face it alone. Join us for an educational program and connect with others who understand what you’re going…
Researchers at Columbia University have found a way to apply CRISPR, a tool used in gene therapies, to new types of disease, reports News Medical. The study was carried out…
A new documentary, Unrest, explores myalgic encephalopathy (ME), also known as chronic fatigue syndrome. The film was premiered at the Sundance Film Festival 2017 where it received an award for…
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
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