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,…
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…
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 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…
Amyotrophic Lateral Sclerosis (ALS) stops communication between the brain and voluntary muscles. Patients slowly lose muscle strength, affecting their ability to walk, talk, and even breathe. The illness eventually results…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
According to a story from Science and Technology Research News, a group of researchers from the Cleveland Clinic led by Dr. Aziz Nazha will soon present a new myelodysplastic syndromes…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
What if you could prevent all of the world's most fatal genetic diseases? According to Lee D. Cooper, there's a way- we're just not educating people on how to do…
While rare disease affects people all around us, often the mainstream press . Which is why it's extra important when a news outlet as big as the Los Angeles Times…
According to a story from sectorpublishingintelligence.co.uk, the clinical stage pharmaceutical company Retrotope recently announced that the first patient had been dosed in its Phase 2/3 clinical trial. The trial will…
The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Patient Worthy. According to a story from Vox, a review of over…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
According to a story from gazettelive.co.uk, Jenni Mawson, who is currently 19 years old, has faced a years long struggle with chronic fatigue syndrome, which is also known as myalgic…
According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD)…
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