According to a story from Globe Newswire, the biotechnology company Wave Life Sciences recently announced that its investigational product WVE-210201 has received both Rare Pediatric disease designation and Orphan Drug…
According to a story from USA Today, despite numerous warning letters sent from the US Food and Drug Administration to the offices of doctors and other medical professionals across the…
Researchers have found that an artificial intelligence (AI) system is able to recommend patient referrals for over fifty eye conditions as accurately as world-leading doctors. The project is part of…
According to the Dover Post, there are a few crucial questions to ask your doctor upon being diagnosed with breast cancer. By asking these crucial questions, patients will be…
The United States FDA has approved the first generic version of EpiPen and EpiPen Jr (epinephrine). The approval was granted to Teva Pharmaceuticals USA, who will now be able to…
Two companies, GtreeBNT and YuYang DNU, will collaborate in a joint venture to develop the investigational drug RGN-137 for the treatment of epidermolysis bullosa. For more details, you can read…
According to a story from news-medical.net, a recent study suggests that a class of drugs called BRAF inhibitors could be an effective treatment option for ovarian cancer patients who carry…
According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…
According to a story from Pharma Times, four new medications were recently approved for coverage on the Scottish NHS by the Scottish Medicines Consortium (SMC). Some of these drugs are…
The US FDA has awarded Breakthrough Therapy Designation to encorafenib (braftoviTM) combined with binimetinib (mektovi®) and cetuximab for use as an investigational (not FDA-approved) treatment of BRAFV600E–mutant metastatic colorectal cancer.…
Gaucher patients in New Zealand are facing a change. New Zealand’s Pharmaceutical Management Agency (PHARMAC) recently proposed a change in their supported Gaucher treatment from Cerezyme to Elelyso. The switch…
According to VeroNews, one of the biggest issues at the retirement community Treasure Coast is malnutrition. For patients with other serious conditions, like chronic kidney disease, the hospital as…
A Biologics License Application for ElzonrisTM (also known as tagraxofusp and SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm has been accepted for filing by the United States FDA.…
According to information provided by the National Hemophilia Foundation, people living with hemophilia now have the opportunity for a greater level of financial support. This new form of aid comes…
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
According to HSC News, researchers at the University of Southern California have engineered a device that may have a sizable impact on those with pediatric hydrocephalus. By troubleshooting the issues…
According to 9News, Harry Campbell was born very premature at 27 weeks. Not only this, but his dimensions were almost unfathomable. Harry was able to fit into the palm of…
The company Epizyme has announced the results of a Phase 2 clinical study of its lead candidate drug, tazemetostat, in the treatment of relapsed or refractory malignant mesothelioma with loss…
An investigational drug called ALT-P7 is being developed as a potential medicine for certain forms of cancer. The drug has received Orphan Drug Designation for the treatment of gastric cancer…
According to a story from Newswise, an organization called the National Comprehensive Cancer Network (NCCN) has recently released treatment guidelines for a rare type of cancer that can affect women…
According to a story from SFGate, many survivors of childhood cancers are plagued with financial burdens for the rest of their lives. This is because treatment of childhood cancer can…
According to a story from The Guardian, a recent study suggests that cannabidiol, or CBD, could magnify the effectiveness of treatment for pancreatic cancer. CBD is naturally found in the…
The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…
According to BioSpace, a new treatment for pemphigus vulgaris (PV) has been recently approved by the U.S. Food and Drug Administration. The treatment, Rituxan (rituximab), is geared towards adults who have…
The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
