According to a story from EurekAlert!, the results of a recent Phase 3 clinical trial suggest that the targeted therapy sorafenib could be an effective treatment option for desmoid tumors.…
The Kids Wish Network (KWN) works to provide positive experiences for children age 3 to 18 who are living with life-threatening conditions. With the help of various sponsors, they are…
According to a story from WebMD, the US Food and Drug Administration recently issued a warning about a class of antibiotic drugs called fluoroquinolones. They have been linked to an…
Batten Disease There are 13 different forms of Batten Disease defined by the medical community. Each presents similarly but there are differences in the severity of symptoms as well as the…
"I feel powerless to help them. There needs to be a change. And change ought to begin in the form of a policy shift." These are Jay Avasarala's words in…
According to a story from Gizmodo, the genetic mutation which causes certain types of dog breeds, such as Boston terriers and bulldogs, to have spiral shaped, corkscrew tails appears to…
According to an article at PR Newswire, Bridge Biotherapeutics Inc., a South Korean biotechnology company, has announced the opening of three of its scheduled ten clinical study sites for the…
According to a story by MarketScreener, pharmaceutical company Acer Therapeutics Inc. has submitted a New Drug Application (NDA) for its vascular Ehlers-Danlos syndrome (vEDS) drug Edsivo. The NDA was granted…
Neuromyelitis optica (NMO), or Devic's disease is a rare autoimmune condition which causes inflammation in the optic nerve and/or spinal cord. The condition affects 5 in every 1,000,000 people and…
Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
Thanks to a Phase 2 trial, pediatric patients with Philadelphia chromosome-positive acute lymphoblastic leukemia or Ph+ ALL, now have a new treatment option. The FDA has announced approval of Sprycel…
It has just been announced that Capricor Therapeutics has put their Duchenne Muscular Dystrophy (DMD) clinical trial on hold. Fortunately, the company does plan on continuing the trial. What is…
According to a story from ESPN, there were times when Lynn Rogers wasn't sure if she was going to be able walk, much less run, ever again, but she wound…
According to a story from the Washington Post, 47-year-old Hector Hernandez of Downey, CA, was subject to frequent jokes about his prominent stomach. Hector has always been a large man,…
PPIs or Proton Pump Inhibitors are one of the top 10 selling generic drugs in the United States. They're used to treat heartburn and acid reflux by suppressing acid production…
Chronic kidney disease (CKD) affects approximately 26 million people in the United States. CKD usually develops after a patient has already battled a different illness which has placed a toll on…
According to a story from clinicaltrials.gov, an observational study titled "The Impact of Charcot-Marie-Tooth Disease" was added to the site on December 20th, 2018. The study itself first began on…
Apellis Pharmaceuticals has just announced that they have received Orphan Drug Designation from the FDA for APL-2, a C3 complement inhibitor. The company is currently investigating the drug as a…
University of Copenhagen researchers have been studying the cause of Huntington’s disease in order to potentially develop a treatment. They have determined that failure of glial cell maturation is a…
Nonalcoholic steatohepatitis (NASH), is condition most commonly caused by obesity or type 2 diabetes. It can result in fibrosis, complications in the lung and cardiac system, liver cancer, liver cirrhosis,…
According to an article originally published by HuffPost, an ambitious 34-year-old doctor is peddling plasma transfusions at steep prices, claiming a number of dubious health benefits. Ambrosia, LLC In 2014…
Paroxysmal nocturnal hemoglobinuria (PNH) prematurely destroys the bodies red blood cells (hemolysis). It can cause severe anemia, kidney disease, fatigue, as well as other symptoms. Up until now, there has…
"It helps to share experiences with others who understand what I am going through." Jill Price, a mom with Charcot-Marie-Tooth Disease talks about how social media has helped create a…
According to a story from ksl.com, a recent poll found that the majority of Americans are in support of using gene editing technology in order to protect newborns from genetic…
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