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…
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A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
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…
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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…
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…
If you are an avid follower of the latest news, then you have probably heard about one the latest sensational headlines to come out of DC lately: the US Department…
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…
Happy Thursday Everyone! We hope everyone's spring is off to a good start! This week, we're highlighting four articles about research updates. We have news pertaining to Methylmalonic Acidemia patients…
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…
According to a story from The McGill Tribune, a little girl named Mathilde Poliquin recently died at the Montreal Children's Hospital. She was only two and half months old and…
According to a story from Myeloma Research News, the UK National Institute of Health and Care Excellence (NICE) has recently decided to approve a three part combination treatment for coverage…
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…
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…
According to a story from fortune.com, the French drug startup Pharnext, led by CEO Daniel Cohen, recently made an announcement not to long ago that made its stock shares rocket…
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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