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…
According to a story from the Tennessean, learning that both her husband and daughter had the rare and dangerous disease atypical hemolytic uremic syndrome (aHUS) was devastating for singer-songwriter Cameron…
According to a story from globenewswire.com, the drug development company Galectin Therapeutics, Inc., recently announced its plans to go forward with Phase 3 trials for GR-MD-02. The company is dedicated…
A new fitness competition held its first contest this April in Sunderland. A contest based around individual functional fitness, First Means Everything donated a percent of its sign-up fees to…
Collaboration between the FDNA, the Cure Sanfilippo Foundation, and the Jonah’s Just Begun Foundation has led to technology that successfully recognises the facial phenotype (observable characteristics) of patients with mucopolysaccharidosis…
It may not be surprising news to find that the United States now pays more for prescription drugs than any other high-income country. This was not always the case however. Read…
The pharmaceutical company Biohaven has announced that they will be using an expanded access program to make an experimental drug for amyotrophic lateral sclerosis (ALS) available to patients before F.D.A.…
In Canada, it is illegal to require a person to disclose results of genetic testing in any form of contract. The government of Quebec, however, recently issued a series of…
A recent study published in the Lancet has investigated the effects of starting chemotherapy on patient survival, according to the UK Government website. It was found that many factors can…
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