According to a story from Angioedema News, a recent clinical trial has demonstrated that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of acute…
According to a story from the Broad Institute, there are still many mysteries to uncover related to inflammatory bowel diseases, which includes the rare form ulcerative colitis. The understanding of…
According to a story from Angioedema News, a team of researchers has used data from a recent study to provide up to date recommendations for diagnosing hereditary angioedema in the…
According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…
According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…
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Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
According to a story from EurekAlert!, in recent years immunotherapy, which are methods of treatment that enhance or harness the body's immune system to fight disease, have become increasingly prominent…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc has announced the release of preliminary topline data findings from its phase 2a clinical trial, which tested…
According to a story from Pulmonary Arterial Hypertension News, data from an open label extension trial of the drug Orenitram as a treatment for pulmonary arterial hypertension has demonstrated that…
According to a story from Fabry Disease News, a recent study has found that despite changes to sperm cells, the fertility of men with Fabry disease is not affected. The…
According to a story from BioSpace, CTI BioPharma Corp. has recently concluded an End of Phase 2A meeting with representatives from the US Food and Drug Administration (FDA). This meeting…
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Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
According to a story from Science Daily, a recent study has found that osteosarcoma, a form of bone cancer, displays similar genetic characteristics when it appears in both humans and…
According to a story from Medical Daily, a recent study found that male Parkinson's disease patients that were sexually active tended to have less severe symptoms and slower disease progression…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
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Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…
According to a story from Huntington's Disease News, Egypt is able to claim the dubious honor of being the Huntington's disease capital of the world when it comes to its…
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia…
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Myelofibrosis (MF) comes under the heading of rare cancer. A recent article in Newswise, University of Utah, describes the disease as the failure of bone marrow to produce normal…
The Study Juvenile idiopathic arthritis (JIA) is a rare disease that causes inflammation in the joints. It is of unknown origin and has no cure. Previous investigations in studies with…
Zogenix has just announced that they have not only finished enrollment in their Phase 3 investigation of Fintepla for Lennox-Gastaut Syndrome (LGS), but that the final patient has been randomized…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
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