According to a publication from health website Stat, a new way of thinking about the price of drugs is taking root in Washington D.C. and beyond. "Value-based agreements" are a…
According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact…
According to a press release from American biotechnology company Soligenix, the European Medicines Agency's (EMA) Pediatric Committee recently agreed to the Company’s Pediatric Investigation Plan (PIP) for experimental oral mucositis…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
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An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…
My first admission is that I actually know quite a bit about ABC's The Bachelor. Yes, that includes The Bachelorette too. I first started watching “ironically“ with my girlfriend, who…
Nigam Shah and Purvesh Khatri, professors at Stanford University, along with graduate student Madeleine Scott have just announced results from a research study which could be pivotal for those living…
Happy Spring! Anyone else got that spring fever? Unless you're getting tortured by allergies, it is a great time to be outdoors. The bees are buzzing, the flowers are blooming,…
Tracheostomies in ALS A tracheostomy is essentially an invasive procedure which helps patients breathe. It also removes secretions from their lungs. Doctors cut an opening into the patient's neck and…
According to a story from Radio NZ, two grandmothers have taken a central role in getting a petition delivered to the parliament of New Zealand. This petition is calling for…
According to a story from CheckOrphan, there are currently very few effective treatment options for chordoma, a rare, slow growing tumor that originates from bone. However, a recent research discovery…
RADOIR Rare Diseases Foundation of Iran (RADOIR) is an organization dedicated to supporting those living with rare diseases in Iran. It is a nonprofit which was founded in 2008 with…
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
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…
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