According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…
According to a story from MD Magazine, the team at Spatial Transcriptomics has utilized an innovative computational approach in order to map gene expression on the spinal cords of patients…
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An article in Austin Gastroenterology recently asks this question. It also confirmed what we already know, that genetic testing is now available to the average person and in fact,…
Optiz C Syndrome Optiz C syndrome (OCS) is an ultra-rare condition whose symptoms are often mistaken for Bohring-Optiz syndrome, Kabuki syndrome, or Kleefstra syndrome. The disease affects patients physical and…
BOS Bronchiolitis obliterans syndrome (BOS) occurs in 50% of patients who endure a lung transplant. In essence, the patient's immune system attacks the bronchioles in the new lung, causing blockages.…
According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…
According to a publication from EurekAlert, a recent study suggests a common, typically symptomless form of the herpes virus called cytomegalovirus may cause faster disease progression in cystic fibrosis (CF)…
According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe in a phase 1/2 clinical…
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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…
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