Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
The Jaiswal family of Lucknow, the capital of Uttar Pradesh state in northern India, knows firsthand that rare diseases are not always that. CheckOrphan has reported that three of his…
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
According to a story from The Oncology Times, the US Food and Drug Administration (FDA) has been moving forward with several treatments and potential treatments for a variety of different…
Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…
According to a story from tonic.vice.com, Liz Aronin and Jamie Mills both thought that everything was going smoothly with their newborn son Quinn, but within a few months, it became…
99% Invisible recently reported on the history of "Orphan Drugs." These drugs are an enormous source of income for pharmaceutical companies - but the explosion in their production is a…
Polycythemia Vera or PV, is a rare blood cancer. It's caused by a mutation which results in the overproduction of red blood cells. Too many blood cells significantly increases your…
According to a story from MarketWatch, Annie Wilson was diagnosed with spinal muscular atrophy when she was only six months old. With no real treatment options available, doctors told her…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a story from prnewswire.com, the drug developer Mirum Pharmaceuticals recently announced that is has entered an agreement with Shire which grants exclusive rights for marketing and development of…
According to a story from ncbnewyork.com, a recent study found that $7 million on donations on crowdfunding sites for medical patients have gone towards treatments that are medically unsound and…
According to a story from Xconomy, the Rainwater Foundation, which was first founded in the 90s by Texas billionaire Richard Rainwater, is offering cash rewards to the tune of $250,000…
According to a story from Buzzfeed News, Paige Rogers was 21 years old when she was admitted to the hospital. She was having trouble breathing because of cystic fibrosis. Over…
Adult Growth Hormone Deficiency (AGHD) is a rare condition when your body produces less growth hormone (GH) than normal for your age and sex. AGHD may affect your body systems,…
According to Biotech and Pharma Industry News, British pharmaceutical manufacturer Mallinckrodt has reported inconclusive results in its Phase 2/3 trial of VTS-270, a drug candidate for the treatment of the…
REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a…
Short bowel syndrome is a malabsorption disorder that causes the body to not get the fluids or nutrients it needs. It can cause dehydration and malnutrition, in addition to other…
Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from tradeshownews.vporoom.com, the biopharmaceutical company Viking Therapeutics, Inc., has recently released the results from a Phase 2 clinical trial. This trial is testing the company's investigational therapeutic…
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