February 16, 2018 Governor Herbert Declarations: Rare Disease Day on February 28th Undiagnosed Rare Disease Day on April 29th Park City, Utah. (February 16, 2018) – Rare and Undiagnosed Network…
Katie Price, a former British model, has accepted the challenge of running the next London marathon with intent to fundraise for IPF, idiopathic pulmonary fibrosis, a disease her mother was…
According to a story from biomedcentral.com, doctors have begun screening infants for tyrosinemia type 1. However, it is still unclear how accurate the screening process for this genetic disorder is.…
According to a story from Specialty Pharmacy Times, medical professions are starting to recognize the need for a new standardized treatment plan for pediatric rheumatic diseases. Pediatric diseases are illnesses…
Little Natalie Essex was diagnosed with spinal muscular atrophy type 2 (SMA) when she was only a year and a half, reports CBC News. It all started when her mother, Meredith,…
Dangerous forms of brain cancer such as glioblastoma are almost impossible to treat. However, new research by Dr. Roel Verhaak could change that. Glioblastoma is the most aggressive form for…
According to a story from EurekAlert, a recent study published in the journal Pediatric Research reveals new protein biomarkers that researchers can use to evaluate how patients with progeria have reacted…
Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…
According to a story from biospace.com, a treatment developed by the pharmaceutical company Pfizer was given Breakthrough Therapy designation by the Food and Drug Administration (FDA). The drug is a…
According to an article from news-medical.net, researchers are beginning to delve into the causes of a rare lung disease called primary ciliary dyskinesia. This research is revealing previously unknown information…
The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…
Just this past week 17-year-old Tina McElligott from Kilflynn, Co.Kerry, passed away from rare disease complications, reported Irish Mirror. She had suffered her entire life with Alpers syndrome, having consistent and intense…
Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…
Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…
Spark Therapeutics will be hosting a conference call this Tuesday. For those interested in the business side of therapy, this is an interesting opportunity to observe. Find out more about…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
Maddie Holt from Everett Washington is a 5-year-old with severe disabilities. She can barely hear or see, and she certainly can't walk or talk, reports Maine Public. Unfortunately for her,…
Andy Trapp is a videographer who runs AB Trapp Productions, a small business documenting weddings and family occasions. He has been living with uncontrolled PKU since the age of 8.…
According to an article from BBC News, Jackie Harrison had a daunting choice to make. There was a fifty percent chance that she would contract Huntington's disease, a rare condition…
According to USA Today, a new study suggests that pregnant women should be conscious of their diet and avoid any restricting diets that include low-carb and gluten-free limitations. The study…
According to a story from the National Hemophilia Foundation, the pharmaceutical company Novo Nordisk announced that its product REBINYN was now available for hemophilia B patients. The treatment is intended…
According to a story from CBS News, Hazel Hammersley was diagnosed with neuroblastoma at age two. As a result, she was forced to fight for her life at a very…
According to a story from Bronchiectasis News Today, the results of a new study suggests that bronchiectasis may trigger autoimmune inflammatory disease. The study found that the condition was frequently…
A severely aggressive brain tumor in 19-year-old Callum Hardyman has forced his brain to turn sideways, reported The Sun. Callum suffers from a terminal brain cancer called glioblastoma. To learn…
The Orphan Disease Center in association with the University of Pennsylvania and the Loulou Foundation just announced their 2018 Pilot Grant Program for CDKL5 Deficiency Disorder. CDKL5 deficiency disorder (CDD)…
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