According to a story from PR Newswire, pancreatic cancer rates in the US are increasing by almost two percent each year. This puts the disease in position to become the…
Juvenile idiopathic arthritis, Lupus, and Malaria are diseases which currently have limited treatment options. But a new discovery by researchers at the Benaroya Research Institute (BRI) could potentially lead to…
According to a story from BioPortfolio, the Bristol-Myers Squibb Company recently announced that the European Commission has recently approved the first combination treatment for renal cell carcinoma available in the…
The Right-To-Try Law is a federal legislation which allows terminally ill patients the opportunity to use experimental drugs that don't yet have FDA approval. It's controversial at best, but some…
Merkel cell carcinoma (MCC) is a form of skin cancer. In the United States, about 2,500 people are diagnosed with MCC each year. The condition is treated with chemotherapy, immune…
FDNA has created a new type of algorithm which may be able to help diagnose rare genetic conditions like Noonan Syndrome by a simple picture of a person’s face. What’s particularly…
According to a story from BioPortfolio, the pharmaceutical company Acer Therapeutics, Inc., recently announced the company's New Drug Application (NDA) has been accepted by the US Food and Drug Administration…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
Happy Thursday! We hope everyone's new year is off to a good start. If you're in need of some inspiration, we're highlighting a story of a woman with CIDP accomplishing…
A recent study conducted in Oregon has shown that emergency medical responders may be biased against minority patients. It's the concept of unconscious bias. Discrimination is illegal, but that doesn't…
The Patient Access Network Foundation (PAN) is an organization which works to ensure that patients living with rare, chronic, or life-threatening diseases are not prohibited from receiving treatment due to their…
"Beyond research motivation, I want to give a voice to the disease to help researchers empathize and further understand who all their hard work is going toward." These are words from…
According to a story from drugs.com, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that is had recently completed a end of Phase 2 meeting with officials from the US…
Prader-Willi Syndrome (PWS) is caused by deleted genes on chromosome 15. This results in improper signaling of satiety, leading to obesity. Individuals with the condition can suffer from respiratory/cardiac disease, gastric…
Myasthenia Gravis (MG) is a progressive autoimmune disorder which results in the weakening of muscles. Most people with the condition are able to manage their symptoms with treatment and have…
When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…
According to a story from WebMD, the US Food and Drug Administration recently issued a warning about a class of antibiotic drugs called fluoroquinolones. They have been linked to an…
Batten Disease There are 13 different forms of Batten Disease defined by the medical community. Each presents similarly but there are differences in the severity of symptoms as well as the…
"I feel powerless to help them. There needs to be a change. And change ought to begin in the form of a policy shift." These are Jay Avasarala's words in…
Neuromyelitis optica (NMO), or Devic's disease is a rare autoimmune condition which causes inflammation in the optic nerve and/or spinal cord. The condition affects 5 in every 1,000,000 people and…
Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
Thanks to a Phase 2 trial, pediatric patients with Philadelphia chromosome-positive acute lymphoblastic leukemia or Ph+ ALL, now have a new treatment option. The FDA has announced approval of Sprycel…
It has just been announced that Capricor Therapeutics has put their Duchenne Muscular Dystrophy (DMD) clinical trial on hold. Fortunately, the company does plan on continuing the trial. What is…
PPIs or Proton Pump Inhibitors are one of the top 10 selling generic drugs in the United States. They're used to treat heartburn and acid reflux by suppressing acid production…
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