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…
Jim and Melea Martin are Outreach Partners with Future by Design, a family support group organized by the Cystinosis Research Network. Melea has cystinosis and Jim is her husband. We…
This article discusses the plot of the biographical film Brain on Fire. Susannah Cahalan was twenty-four when she began experiencing seizures, hallucinations, and, after only a few weeks, catatonia. Her…
Tomorrow is World Scleroderma Day! This date is an opportunity for anybody with an interest in scleroderma to join together as one united voice to shine a spotlight on a…
According to a story from today.com, Dr. David Fajgenbaum was first diagnosed with Castleman disease in 2010. His first encounter with the illness was terrifying. He was confined to the…
Visit any pediatric ward in a hospital or medical center and you will see many pieces of medical equipment, but visit the OT and PT departments and you are in…
According to Sleep Review, researchers at the University of North Carolina School of Medicine are studying DNA repair after damage from anti-cancer drugs as well as DNA repair and how…
Although having a child with disabilities is no doubt stressful, the stress and worry does not necessarily lessen as the child grows older. As children with disabilities age towards adulthood…
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…
Andrea Jenkyns is a member of the British Parliament and new mother. She was elected as the Conservative Member of Parliament for Morley and Outwood at the 2015 general election.…
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 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 medicalxpress.com, Hepatocellular carcinoma is the third leading cause of cancer deaths in the world. It is also the most common form of liver cancer. New research from a…
Arrowhead Pharmaceuticals, Inc. have announced that they have completed patient enrollment for a Phase 1 clinical trial investigating ARO-AAT, an investigational therapy for the treatment of a genetic liver disease…
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…
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