No matter which way you spin it, Ehlers-Danlos syndrome (EDS) is generally not a great time. EDS is a rare genetic condition that affects connective tissue throughout the body. It…
A class of drug called proton pump inhibitors (PPIs) that are used to treat acid reflux and heartburn have been linked to an increased risk of stomach cancer for some…
According to a story from Medical Xpress, a recent study is revealing pronounced differences in the brains of people with fragile x syndrome when compared to the brains of people…
According to a story from ankylosingspondylitisnews.com, a significant proportion of patients stopped taking a biosimilar for infliximab within six months of transitioning to the new medication. This suggests that the…
According to a story from The Irish Times, a new campaign is encouraging people with the rare lung condition idiopathic pulmonary fibrosis to keep exercising. The campaign is centered around…
A drug being developed to treat cystic fibrosis has just been granted Fast Track Designation by the US Food and Drug Administration (FDA), reports PR Newswire. This shows that previous…
According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…
Tyler Perry’s Madea may have saved Boone Begner’s life. Born with heterotaxy syndrome, Boone’s heart works with only three chambers instead of four. The condition often becomes extremely dangerous to…
According to a story from wave3.com, time was running out for Leyan Albazzour, who is nine years old. Born in the Palestinian city of Ramallah, the treatment for her Apert…
A recent story from Science Daily is highlighting just how difficult it can be for patients with rare diseases to get an accurate diagnosis. For patients with mitochondrial disease, the…
A new study looking at a test used to diagnose narcolepsy has found that it often produces inconsistent results for the same patients, reports Sleep Review. This highlights a current…
Recent news from Apple shows a new series of emojis coming with the next iOS update. The set intends to “better represent individuals with disabilities.” To accomplish this, Apple partnered…
In some cases of multiple sclerosis, patients report a decrease in their sense of smell. This by itself is not unusual given the nature of the disease. A small study…
According to a story from Neurology Advisor, a retired physician experienced the challenges of being a rare disease patient first hand after being diagnosed with primary periodic paralysis thanks to…
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA).…
Peter Kay, a British comedian and actor, organised a screening of four episodes of his hit TV series Car Share in order to raise awareness and donations for a charity…
Major pharmaceutical company Pfizer recently announced the success of a new experimental product. The drug targets a disease called transthyretin cardiomyopathy, related to amyloidosis, a rare and dangerous disease that affects…
According to a recent story from CityNews Toronto, Alara Ozkose's Dravet syndrome was preventing her from being able to live a life that was even close to normal. She was…
A new drug called Altemia is being developed to treat sickle cell diseases in children, reports BioPortfolio. It has recently been awarded Orphan Drug designation in both the United States…
Researchers at Northern Arizona University (NAU) are using microbial sequencing to better understand the causes of airway inflammatory diseases, including chronic rhinosinutsitis and cystic fibrosis. They hope that by studying…
A potential treatment of pulmonary infection for patients with cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD) has just been awarded U.S. patent status, reports Medicalxpress. Dr Daniel Hassett, a researcher…
According to a story from Newswise, researchers at the University of Texas Southwestern Medical Center are reporting the a new drug developed for the treatment of rare, pediatric cancers, has…
Researchers at Northwestern University (NU) have published research showing that changes in the brain causing spinocerebellar ataxia type 1 (SCA1) occur at a younger age than previously thought, reports NeuroscienceNews.…
According to a story from the Seattle Times, a massive $150 million gift from Richard and Susan Rogel to the University of Michigan is slated to go towards the school's…
According to a story from mrknewsroom.com, the pharmaceutical companies Merck and AstraZeneca recently announced that the marketing application for the investigational drug olaparib (brand name Lynparza) was recently certified for…
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