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An encouraging article recently published in Sickle Cell Anemia News announced that due to the urging of pharmaceutical companies and patient advocacy groups, the FDA has produced an updated draft…
Any fans of RuPaul Drag Race or the art of drag itself? Raise your manicured hand! Well, regardless of your answer - watch how the world of drag and rare…
GT Biopharma has just announced that their Phase 1 clinical trial for Mastocytosis, Myelodysplastic Syndrome (MDS), and Acute Myeloid Leukemia (AML) has been authorized to begin. Although this investigation had previously…
A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…
"When only 100 people in the world share your child’s specific gene mutation, it is hard to find anyone who understands, including doctors." Rare diseases are at a disadvantage when…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
According to a story from EurekAlert!, a team of scientists affiliated with the UCLA Jonsson Comprehensive Cancer Center have been conducting research that has revealed a new potential therapeutic approach…
According to a story from VC Reporter, Dr. Sabine Hazan-Steinberg is a true believer in the medical capabilities of fecal microbial transplant (FMT). Despite the fact that this procedure has…
According to a story from BioPortfolio, the biopharmaceutical company AVEO Pharmaceuticals and the lung cancer diagnostic company Biodesix recently released the results from the Phase Ib clinical trial testing a…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company's application to…
According to a story from Market Screener, the biopharmaceutical company Imbrium Therapeutics LP recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation for the…
According to a story from andreeger.com, gene therapy, a type of medical treatment in which genes are modified in order to treat disease, has become a hot button topic in…
According to a story from businesswire.com, the biopharmaceutical company PellePharm, Inc. recently announced that it has dosed the first patient in the company's Phase 3 clinical trial. This trial is…
According to a story from EurekAlert!, a recent research study has found that a two part immunotherapy combination treatment has the potential to be a useful treatment for high-grade neuroendocrine…
Recently Dr. Janet Maynard, who currently is the director of the US Food and Drug Administration's (FDA) Office of Orphan Drugs Development, wrote an article in which she discusses the…
Happy April! We hope everyone's month is going well! This week, we're highlighting two patient stories and two research stories. First, we have the story of Carolyn, a pemphigus patient…
According to a publication from World Pharma Today, the 16th annual BioAsia conference saw the greatest ever amount of participation at what is consistently Asia’s largest biotechnology and life science…
At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…
Nonalcoholic fatty liver disease (NAFLD) is a condition in which the buildup of fat in the liver poses health concerns. Nonalcoholic Steatohepatitis (NASH) is a more severe, and more rare…
If you haven’t heard of bullous pemphigoid, you’re not alone, the condition isn’t typically well known even by physicians. For those living with it, this tends to mean a long,…
According to a publication from FOXBusiness, Waltham, Massachusetts-based Thermo Fisher Scientific Inc. has agreed to buy out Brammer Bio for $1.7 billion. With the acquisition, Thermo Fisher, a lab equipment…
According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia. Frontotemporal Dementia Dementia is a blanket term…
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Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
According to the publication from RedlionTrader, a second man infected with HIV seems to have been cleared of the infection, those scientists remain highly cautious of touting the case as…
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