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…
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…
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 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…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…
A $1.4 million dollar donation to the University of Massachusetts Medical School will help to fund research into a gene therapy for Tay-Sachs disease as it transitions from pre-clinical to…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
According to a story from Scleroderma News, a recent study found that men were more likely to have severe scleroderma early in their diagnosis in comparison to women. This is…
Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the…
A recent study has looked into the use of video visits with patients by clinicians in Sweden, to try to find out how doctors decide which patients to offer them…
According to a story from NRI Pulse, Kiran and Anju have been forced to watch over the past year as their daughter Aadya's physical and mental condition declined. After serious…
According to a story from Futurity, a recent study has identified a protein that plays a central role in the onset of a rare cancer called subcutaneous panniculitis T lymphoma…
In a recent news article, the Northwestern Medicine Feinberg School of Medicine has shared details about an experimental device that may have the potential to dramatically improve the treatment of…
An investigational new drug for bronchopulmonary dysplasia, called CF-MEV-132, has completed pre-clinical in-vivo testing. According to The Cell Factory, who are developing the drug, the potential treatment is now ready…
According to a story from EurekAlert!, a recent study from the Children's National Health System has found that some cystic fibrosis patients are not getting sufficient doses of antibiotics in…
BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…
In 1993, researchers found one of the causes of familial amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder which affects the motor neurons. Motor neurons are responsible for transmitting…
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