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…
Andrew Macnab received a diagnosis of dermatomyositis last year. His sister Helen Keith is preparing for her first marathon in order to help Andrew. The money Helen raises will go…
A California family has lost 12 family members to the newly discovered rare disease known as hereditary sensory and autonomic neuropathy type 1E (HSAN 1E), which involves a mutation on…
Senator John McCain has been fighting for most of his adult life. Now is no different. He faces a battle with brain cancer. His daughter, Meghan supports him often in…
The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…
Researchers have found a new way to monitor disease progression in people with Charcot-Marie-Tooth disease type 1 (CMT1), reports Charcot Marie Tooth News. CMT1 is an inherited genetic condition that…
Here's something on the lighter side of disease prevention efforts! Earlier this month, the Centers for Disease Control and Prevention tweeted a photo of a poppy seed muffin which was meant…
Some people would describe Susan Hutchinson as a thrill seeker. She's type of person to climb volcanoes. She joined the army reserves. Perhaps that’s what makes a diagnosis like chronic…
May is national Cystic Fibrosis Awareness Month! Cystic fibrosis (CF) is a genetic condition that causes progressive respiratory and digestive system damage. Someone with CF has thick, sticky mucus, versus…
According to a story from Science Daily, a recent study found that a rigorous exercise program reduced fatigue in testicular cancer patients and helped boost their self-esteem. Patients with the…
A pre-clinical study of a treatment for Rett syndrome has produced encouraging results, and it is hoped that this will lead to further research. The full story can be read…
Cushing's syndrome may be due to adrenocortical carcinoma, a tumor of the adrenal gland. This rare tumor can cause increased hormone levels in patients that are commonly associated with Cushing's…
According to a story from Medscape, the House of Representatives recently voted to pass a "right to try" bill. The bill they voted on was previously approved by the Senate…
The Alagille Syndrome Alliance (ALGSA) is preparing to host its first annual gala, The Gala of Dreams, this year on Saturday, June 30th, 2018 at the San Diego Marriott Del…
According to a story from CBS Minnesota, legislation was recently filed in Minnesota that could end the practice of step therapy in the state. Step therapy is a common money…
For a long time, researchers thought that chronic liver inflammation was the driving force of cancer, because the inflammation allows the cancer cells to divide, preventing them from dying. But…
A fourteen-year-old girl called Kira Noble from Edinburgh, Scotland has raised £340,000 (about $460,000) for surgery to remove her neuroblastoma tumour, reports The Daily Record. Kira was first diagnosed with…
According to a story from Cambridge News, Ethan Monk's Williams syndrome managed to evade detection until just two years ago. Ethan had some peculiar symptoms, such as delayed development, unusual…
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