Idiopathic pulmonary fibrosis (IPF) is a rare condition with no known cause and no effective treatments besides a lung transplant. Current therapies focus on slowing progression of the disease but…
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…
The FDA has just announced that they've approved Mayzent for the treatment of relapsing multiple sclerosis (MS). Multiple Sclerosis Multiple sclerosis is a neurological autoimmune disease. It causes a wide…
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…
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…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
A New Partnership Patient Worthy is honored and excited to announce our new partnership with The B.L.A.I.R. Connection. The B.L.A.I.R. Connection was founded by Grey Chapin who knows firsthand what…
A recent generous donation from the Michael J. Fox Foundation for Parkinson's Research (MJFF) will help develop a novel piece of technology which could accelerate the research of new treatment…
As the field of medicine has continually advanced, more and more children (now approximately 80%) are surviving cancer diagnoses. It's amazing. It also means that researchers are able to focus…
Robyn Smyth is a 14-year-old girl from Ireland who was diagnosed with neuroblastoma when she was three. Since then she's undergone countless rounds of chemotherapy, investigational treatments, a treasured period…
A recent article by Joe Graedon compares the FDA and the way the US investigates drug safety to the Federal Aviation Administration (FAA) and the National Transportation Safety Board (NTSB).…
Acute Myelogenous Leukemia Acute myelogenous leukemia (AML) is a rare blood cancer whose prevalence increases with age. There are still extremely limited treatment options for AML and the five-year survival…
A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…
Bruce Alan Fries, President of the Patient Centered Care Advocacy Group, along with Holly Ahern and Chris Fisk from the Lyme Action Network, have issued an official "Information Quality Request…
A preliminary study has found that dementia may only affect select areas of the brain in the early stages. Through this research, they've also uncovered what they believe is a…
The longer a patient sees a doctor, the more their initial trust, confidence, and comfort with them grows. This is amplified for patients living with rare conditions as doctors visits…
When one rare disease is found to be in some way similar to another rare disease, research can often be accelerated for both. For instance, recent discovery by a professor…
Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…
Extremely positive results have just been announced from a 5-year follow-up study of a systemic juvenile idiopathic arthritis (sJIA) treatment called Kineret. This treatment has been approved for use in…
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