According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…
According to a story from pm360online.com, the U.S. Food and Drug Administration (FDA), has given Priority Review status to eltrombopag (marketed as Promacta) after impressive clinical trial results. Eltrombopag was…
Fresh off of Memorial Day, there is no better documentary to watch right now than HBO's John McCain: For Whom the Bell Tolls. The Arizona senator announced last July that he…
According to a story from CNN, substitute teacher Cynthia Tisdale was widely admired by her family and friends. Cynthia was murdered in the Santa Fe High School shooting on May…
According to a story from kait8.com, the company Harmony Biosciences, Inc., has recently announced that the U.S. FDA has given Fast Track and Breakthrough Therapy designations for its experimental therapy…
According to a story from apnews.com, sickle cell disease gets very little research or funding despite that fact that about 100,000 people in the U.S. are affected by it. The…
The not-for-profit healthcare organisation Sanford Health is planning to implement routine genetic testing for patients to identify a patient’s risk for a range of rare, and more common, diseases. The…
According to a story from PR Newswire, the pharmaceutical company BioMarin Pharmaceutical Inc. has recently announced that the U.S. Food and Drug Administration as issued approval for their product pegvaliase-pqpz, which…
According to a story from polygon.com, Microsoft has developed a new Xbox Adaptive Controller that is tailored towards players that have reduced use of their hands and fingers. While there…
TGIF, everybody! This week we're sharing music videos by a singer who raises awareness for aHUS, a journey of a dysautonomia patient, and a story about a man who was…
According to a story from inhomelandsecurity.com, military veterans are roughly twice as likely to get amyotrophic lateral sclerosis (ALS), a lethal degenerative disease that kills in as little as two…
May has been designated Behçet's disease awareness month - but unlike so many of the rare diseases we cover, there are not any advocacy organizations dedicated to raising awareness and support…
A new treatment for cystic fibrosis has received a lot of media attention in the UK after the National Health Service (NHS) chose not to fund it due to its…
According to a story from the Washington Post, an outbreak of Nipah virus has claimed a total of ten lives so far in south India. Medical teams are starting to…
According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
Here's something on the lighter side of disease prevention efforts! Earlier this month, the Centers for Disease Control and Prevention tweeted a photo of a poppy seed muffin which was meant…
May is national Cystic Fibrosis Awareness Month! Cystic fibrosis (CF) is a genetic condition that causes progressive respiratory and digestive system damage. Someone with CF has thick, sticky mucus, versus…
According to a story from Medscape, the House of Representatives recently voted to pass a "right to try" bill. The bill they voted on was previously approved by the Senate…
According to a story from CBS Minnesota, legislation was recently filed in Minnesota that could end the practice of step therapy in the state. Step therapy is a common money…
A fourteen-year-old girl called Kira Noble from Edinburgh, Scotland has raised £340,000 (about $460,000) for surgery to remove her neuroblastoma tumour, reports The Daily Record. Kira was first diagnosed with…
According to a story from globenewswire.com, the drug development company Galectin Therapeutics, Inc., recently announced its plans to go forward with Phase 3 trials for GR-MD-02. The company is dedicated…
It may not be surprising news to find that the United States now pays more for prescription drugs than any other high-income country. This was not always the case however. Read…
In Canada, it is illegal to require a person to disclose results of genetic testing in any form of contract. The government of Quebec, however, recently issued a series of…
Scientists working at the Broad Institute of MIT and Harvard are trying to build a collection of every drug ever developed. They are planning on using the collection to re-purpose…
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