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…
According to a story from Dr. Hooman Noorchashm, the US Food and Drug Administration (FDA) has a reasonably effective system for regulating and approving new drugs while appropriately evaluating them…
According to an article originally from WCPO Cincinnati, a woman with fibrodysplasia ossificans progressiva is filing a lawsuit against officials of Ohio's Hamilton County. Jessica Scully, 57, alleges that the…
A story from Family Features Editorial Syndicate, published on PR Newswire discussed how getting diagnosed with a chronic, rare disease can be an overwhelming time for a person. While the experience can also give a meaningful…
Happy New Year! For our first Editor's Choice of 2019, we want to highlight four articles from the last couple weeks. First, we have the story of young man who…
Kinetic Kids is a nonprofit which was originally formed to keep children who needed physical therapy active outside of their actual physical therapy sessions by giving them the opportunity to…
Urea Cycle Disorders (UCD) are rare genetic conditions caused by an enzyme deficiency. The urea cycle is responsible for removing excess levels of ammonia from the body. Individuals with UCD…
According to the Shillong Times, a study published in the New England Journal of Medicine has reported intriguing results when treating sarcoidosis with an already-approved rheumatoid arthritis medication. What is…
According to a story from the Irish Examiner, 31 year old Orla Tinsley is celebrating her first year of life following her life saving double lung transplant operation. This lung…
According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently announced that the first patient has been treated in the company's Phase 3 clinical trial testing sparsentan as…
According to a story from CBS News, Garland Benson, age 14, has undertaken a daunting task in order to save the life of his older sister Christiane. She was born…
Lichen sclerosus is a rare, under-researched, and poorly understood condition. The lack of knowledge concerning the disease has led to a lack of awareness, late diagnosis, and a limited amount of…
A Fanconi Anemia (FA) investigative gene therapy now has PRIority MEdicines (PRIME) designation from the EMA. This designation helps to accelerate the development of therapies for patient populations who have…
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For a long time there have been no treatments for Friedreich's ataxia. With no FDA approved therapies, people with this disease have only been receiving symptomatic treatment. Exicure has changed…
What started as a peculiar and insatiable thirst for water led to a rare disease diagnosis for a Denver family. Although they weren't expecting this challenge, they're ready for the…
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