Mission: Cure is a coalition of patients, researchers, doctors, and entrepreneurs who have taken a new approach to curing diseases. Their first focus is pancreatitis, and they have created a…
Recently, biopharmaceutical company Acceleron Pharma Inc. ("Acceleron") announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic…
Part of the National Institutes of Health (NIH), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and SteroTherapeutics have entered into a Cooperative Research and…
Transitions to adult care for patients who have a chronic respiratory disease, like cystic fibrosis, or a type of neuromuscular disease that influences the respiratory system is something that the medical…
A study published in Neurology has discovered a way to improve autoantibody testing in myasthenia gravis (MG) patients. An issue that physicians and patients face is false-positive results for neuromuscular or autoimmune…
The American College of Rheumatology presented new findings on November 5, 2020 at their virtual conference. They found that in patients with ANCA-associated vasculitis (AAV), hypothyroidism is associated with positive…
For the entirety of 2020, our lives have been dominated by COVID-19, the novel coronavirus linked to 64.5 million cases and 1.49 million deaths across the globe. In the United…
Mesentech and CureDuchenne have announced a partnership that includes generous funding from the Charles H. Hood Foundation. The partnership has developed a targeted therapy for reversing bone wastage in boys…
According to a story from ANCA Vasculitis News, a recent study has determined that monitoring changes in autoantibodies targeting the PR3 protein is useful for predicting ANCA vasculitis remission after…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
Gut bacteria has been known to play a role in a number of conditions, such as inflammatory bowel disease, rheumatoid arthritis, and more. A new study has discovered a connection…
We all know that diagnosing cancer earlier means better outcomes for patients. It’s also essential to monitor the spread of cancer in already diagnosed patients. If we can watch as…
The FDA has recently approved DANYELZA for the treatment of neuroblastoma in pediatric patients over the age of one and adult patients affected by refractory or relapsed neuroblastoma. This approval…
According to a press release from Inovio Pharmaceuticals, the biotechnology company has officially dosed the first patient in its phase 1/2 clinical trial, which will test the company's investigational therapeutic…
Michael George and his team from the University of Pennsylvania have recently uncovered new insights regarding autoimmune patient’s comfortability with care during COVID-19. They've been working since the pandemic began…
In 2019, Japan approved the use of EVRENZO (roxadustat) for patients with anemia of chronic kidney disease (CKD) who were also on dialysis. However, the orally administered therapy was not…
This past November, nearly 9,000 attendees showed up for the American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting. During the meeting, biopharmaceutical company Kodiak Sciences Inc. ("Kodiak") announced that…
When many people hear CAR T-cell therapy, they think about a treatment designed to address cancer. Recently, a new study done on mice models has suggested that an experimental form…
HAE Junior is a Czech-based patient organization aiming to improve the quality of life of children & youth with hereditary angioedema (HAE). It was set up in December 2019 in…
WCVB NewsCenter 5 recently published a news item featuring Nancy and Paul Burke and their three daughters. The Burke family has been living in the shadows of the deadly Sanfilippo…
According to HCP Live, University of Lübeck researchers recently connected rheumatoid arthritis (RA) and pemphigus, an autoimmune disorder characterized by blisters on the skin and mucous membranes. In fact, researchers…
According to a story from Newswise, the ongoing coronavirus/COVID-19 pandemic has become the subject of wild speculation. In our divided nation, many people have chosen to hear what they want…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In a press release by the Parkinson’s Foundation, the non-profit has announced an exciting collaboration with Sanofi, the global pharmaceutical company, which will dedicated $1 million to the nonprofit to…
Recently, scientists and researchers from the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute worked to create an innovative solution for patients with cancerous…
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