BridgeBio Pharmaceuticals has recently announced that their drug, infigratinib, has received the Fast Track and Orphan Drug designations from the FDA. This drug is meant for the treatment of…
According to a story from globenewswire.com, the biopharmaceutical company SELLAS Life Sciences Group, Inc. just announced that it has started screening participants for its phase 3 clinical trial. The trial…
The discovery of a mutated gene is shedding light on the severity of kidney disease in those with Joubert syndrome. The BSND gene is called a modifier gene, and…
Medical science made some great breakthroughs in 2019 and the worldnewspulse.com shares just a few of the most impressive captured moments for science and real life patients. Here are three…
According to a story from the Houston Chronicle, a five year old boy named Myles Cheatham was admitted to the Spring Clinic two days after experiencing symptoms of what was…
According to a story from labblog.uofmhealth.org, one of the defining features of Prader-Willi syndrome, a rare disorder, is hyperphagia, which is extreme overeating and obsession with food. While this is…
Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
The Study: MRx0518-I-003 MRx0518-I-003 is a trial investigating MRx0518 as part of a combination treatment for pancreatic cancer, used with preoperative radiotherapy. The trial will evaluate the therapies safety and…
Sickle Cell Disease Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…
According to a story from ALS News Today, the nation of Sweden is starting to emerge as a leader for amyotrophic lateral sclerosis (ALS) research in Europe. This is mostly…
Galactosemia Galactosemia is a rare disease which affects the metabolic system. It causes the body to not be able to process galactose properly. Galactose is a simple sugar present in…
There have been many defining factors throughout the past decade, especially in the healthcare industry. These changes, both good and bad, have defined this industry. From the opioid epidemic…
According to a story from Pulmonary Hypertension News, the results of a recent mouse study has produced some remarkable results. The study found that melatonin, a naturally occurring hormone in…
According to a story from the University of Aberdeen, 57 year old Sandy Patience of Inverness, Scotland, is one of a total of 801 Huntington's disease patients from around the…
When Jessica Karnes was a kid, she was told she was no longer allowed to eat chicken nuggets. As a self-proclaimed lifelong picky eater—and a four-and-a-half-year-old at the time—this was a…
Oliver Sacks is beloved for his best-selling books that detail case studies of rare neurological disorders. Sacks created a niche when he combined his work as a neurologist with whimsical…
Since April 2019, I have been actively seeking out adults (18+) who are willing to publicly share their stories about their lives with rare and chronic diseases. I feel it…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved avapritinib (marketed as AYVAKIT) as a treatment for gastrointestinal stromal tumors in cases when…
According to an article from the JAMA Network, the role of the human microbiome in our health is just beginning to be understood, and the manipulation of the human gut…
Sandy Patience is a 57-year-old man from Inverness, Scotland, and he has Huntington's disease. His mother and sister also have this disease, so he has seen others who are affected…
According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…
According to a story from OMRF, things were looking dire for Tonja Martin after she was diagnosed with multiple sclerosis. This happened to her in 2010, and she promptly began…
Happy Thursday! We're back with a batch of new article! This week, we have another article from PW contributor, Tom Seaman, who uses his experience with dystonia to help people…
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The biopharmaceutical company Cellectar Biosciences recently announced that the FDA granted its lead phospholipid drug conjugate, CLR 131, Orphan Drug Designation(ODD) for treatment of lymphoplasmacytic lymphoma. ODD is granted by…
AI Therapeutics is a biopharmaceutical company that has created an artificial intelligence platform that is meant to match drugs to new indications. This platform, called Guardian Angel, has matched…
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