According to a story from kait8.com, the drug development company Asceneuron SA recently announced that its experimental molecule ASN120290 was recently granted Orphan Drug Designation by the FDA. ASN120290 is…
According to a story from Scleroderma News, the results from a recent study suggest that the six minute walk test can accurately predict outcomes for patients with systemic sclerosis. A…
The organisations Guerbet and IBM Watson Health are collaborating to develop an artificial intelligence tool designed to support the diagnosis and care of liver cancer. The source article can be…
Put Europe on your calendar for 2018! Usher patients, researchers and family members welcome to the two-day Symposium in July. For more information, contact [email protected]
According to a story from the U.S. Food and Drug Administration (FDA), Commissioner Scott Gottlieb has released a press release which discusses in detail the agency's efforts in the development…
While there have been well-known racial disparities in the diagnosis and survival rates for breast cancer patients, there may be other factors besides the actual race of a patient causing…
At 16 years old, I was told I would need a liver transplant within ten years. Being told at any age that you will eventually need a liver transplant is…
According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…
According to a story from PBS, the FDA has been approving new treatments at a noticeably faster rate than it has in the past. As a result, it appears that…
Happy Friday, everyone! We hope everyone's drinking water, wearing sunscreen, and taking a little time to relax this summer. As we get ready for the weekend, we've gathered four recent…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
According to a story from NPR, plastic straw bans have been gaining traction across the U.S. One of the most shocking examples of planetary degradation as the result of human…
According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…
According to a story from pm360online.com, a new study of Prader-Willi syndrome will soon be under way thanks to a recent collaboration between the biopharmaceutical company Zafgen and the Foundation…
Duncan Avery is father to two children, Kalea, and Noah. At age six, doctors discovered Kalea had a tumor in her brain. They would successfully remove it via surgical methods.…
According to a story from medicalxpress.com, early tests of a gene therapy for X-linked myotubular myopathy are showing promising results. This could represent a major breakthrough for this disease, which…
Happy Independence Day Weekend! As the firework shows wind down, we want to wrap up the week with three great contributor stories, and one research update. First, we have some…
In 2001, when I was three years old, my dad was diagnosed with Adrenoleukodystrophy (ALD). ALD is a rare genetic neurological disorder with no effective treatment or cure. Over the…
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 Check Orphan, a group of scientists from Stanford have begun the process of developing a new test that could help patients with rare Fanconi anemia…
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…
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 news-medical.net, a group of scientists from the Cincinnati Children's Cancer and Blood Diseases Institute have recently published a study in the journal Nature Cell Biology. This…
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