According to UPI, a recent study has found that standard Parkinson's disease treatments may be responsible for a plethora of other concerns as well. Most notably, those who take Parkinson's…
According to Business Wire, a pharmaceutical company, Aurinia Pharmaceuticals Inc., recently announced that they have enrolled the first lupus nephritis patients in the AURORA 2 extension study. Not only this, but Aurinia…
According to a report by the Syndney Morning Herald, Australia may soon approve an in vitro fertilization technique known colloquially as the three-parent technique. This would allow families to greatly…
According to a story from inpublic.globenewswire.com, the U.S. Food and Drug Administration has recently transferred its Orphan Drug designation for dovitinib from to Oncology Venture (OV) from Novartis, according to…
According to Queenstown Research Week, AWCBR, and QMB will be hosting a satellite event in August. The event will center on rare brain diseases, bringing together a group of dedicated…
According to a story from reuters.com, the U.S.-Israeli company BrainStorm Therapeutics will not provide its investigational stem cell treatment under the recently passed U.S. Right to Try law. The company…
The Australian government will give $33 million to researchers to study rare cancers and rare diseases. The original article can be read here, at the Department of Health’s website. Background…
According to a story by CBS News, an unsuspecting virus may become a potential treatment for brain cancer. The study showed that those who were given a genetically modified version…
Happy end-of-June, readers! A lot of people are getting ready for the fourth of July right now. However, as a lot of the rare community knows, not everyone is able…
According to a story from Reuters, the drug development company Merrimack Pharmaceuticals decided to abort its development of a new treatment for pancreatic cancer. The company chose to let go…
According to a story from globenewswire.com, the company Revive Therapeutics, which is dedicated to the development of treatments derived from cannabis and cannabinoids, recently announced that the FDA has decided…
According to EurekAlert! Science News, researchers have pinpointed the certain immune cell that further inflames a heart attack. The immune cell, however, comes with good intentions of trying to fix…
In April, Congress approved the 2018 budget for the Department of Defense (DoD). Included in the DoD is a sizable portion dedicated to the investigation and development of new and…
According to a story from Fierce Pharma, the National Institute for Health Care and Excellence (NICE) has decided to reject the drug Crysvita for coverage on the National Health Service…
A pie to the face?! Yes, you heard right! Saturday, June 30th is Arthrogryposis Multiplex Congenita (AMC) awareness day! It is our duty here at Patient Worthy to shine a light…
According to a story from uk.news.yahoo.com, when Billy Caldwell, age 12, and his mother returned from traveling to Canada, the Home Office initially confiscated a substance that had been the…
According to a story from Human Rights Watch, stories of preventable fatalities due to medical emergencies in immigrant detention centers in the U.S. have begun to emerge. In many cases,…
According to a story from the Washington Post, the US Food and Drug Administration (FDA) has cleared the approval of Epidiolex®, a solution whose active ingredient is a refined version…
According to a story from BioPortfolio, the biotechnology company Vertex Pharamceuticals, Inc. recently announced that the long term use of its innovative cystic fibrosis treatment Orkambi will be covered in…
According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced…
According to a story from Angelman Syndrome news, Angelman syndrome was recently given its own disease unique disease code: Q93.51. The new code will help make researching and tracking the…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
According to a story from CheckOrphan, Express Scripts has prepared a Rare Conditions Care Value (RCCV) program. This program is designed to help make the diagnosis and treatment process for…
Happy start-of-summer everyone! We're kicking off the summer with a wrap up on rare disease news this week. We have an article about a systemic mastocytosis study, and a clinical…
According to a story from The Spectator, Madeline Pitts, who has two forms of terminal cancer, will have June 9th be dubbed "Maddie Day" in her honor. She is thirteen…
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