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…
Living with a rare disease provides enough challenges on its own. For residents of Chicago, however, challenges grew even more difficult as pharmacies shut down one after another. Certain areas…
Researchers have developed a new screening test for Pompe disease that is more sensitive than other options, says a report from January. Pompe disease is a muscular disorder caused by…
According to a story from the Independent, father Nick Sireau is worried that Brexit, the United Kingdom's plan to withdraw from the European Union, could affect the ability of his…
An organisation called PatientsLikeMe that helps to represent patients to governments and pharmaceutical companies has published a press release about the results of two recent studies designed to define and…
According to an announcement from the pharmaceutical company Retrophin, Inc., the company is starting to move forward with phase 3 trials for its drug candidate sparsentan. The drug is in…
According to a story from biopharmadive.com, the pharmaceutical company Roche is reporting a total of five deaths of patients with hemophilia A that have taken the company's product Hemlibra. While…
According to a story from 4-traders.com, the pharmaceutical company SteroTherapeutics recently announced that its drug candidate ST-002 was awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).…
According to a story from rte.ie, a team of scientists from the Trinity College Dublin have managed to discover a new technique that allowed them to shut down inflammatory response…
Happy Friday, everybody! This week we have a story about a woman with Dysautonomia and a wide range of interests, a mother who finds strength in a moment of grief,…
The Health eHeart Study was released on March 21st at the JAMA Cardiology website. The study investigates wearable technology and its ability to detect a common heart rhythm irregularity called atrial fibrillation (AF).…
Today is Bohring-Opitz Syndrome (BOS) Awareness Day! You'd be forgiven if you haven't heard of this rare disease, which is why awareness days exist! Bohring-Opitz Syndrome is an extremely rare condition…
According to a study published in the journal Nature Communications, a very common form of ovarian cancer may actually find its beginnings in the fallopian tubes, the tube that eggs…
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