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…
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