Lennox-Gastaut syndrome (LGS) is a severe and rare form of epilepsy, which causes patients to often have multiple seizures a day. It generally appears in early childhood, and is difficult…
Hereditary hemochromatosis is a genetic form of a condition in which the body builds up too much iron. To read more about hemochromatosis, click here. La Jolla Pharmaceutical Company has…
Severe combined immunodeficinct (SCID) is a rough diagnosis, that leaves the immune system of a patient at high risk for serious infections. Orchard Therapeutics wants to develop a treatment SCID…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
Happy holidays to the Vallecalle family, who are rejoicing in the fact their baby girl is cured of the rare disease, hyperinsulinism. Not only that, but they finally get to…
An amazing 6-year-old boy from Bradford, who was diagnosed with a rare heart condition called LEOPARD Syndrome, creatively started a fund raising campaign revolved around Christmas and singing to raise…
London's renowned Great Ormond Street Hospital (GOSH) has hit a milestone with the construction of the Zayed Centre for Research into Rare Disease in Children. The building just recently reached its…
Everyone with Facebook surely remembers when people were posting videos of themselves having ice cold water dumped on their heads. But, does everyone remember what it was for? The Ice…
Reported by The Washington Post, six years ago Randall Jordan-Aparo was found dead after being sprayed down with a debilitating chemical when wrongfully placed into an isolation cell. According to…
Earlier this month, researchers at the University of Montreal found an encouraging way to improve treatments to cystic fibrosis patients. They discovered key molecules, known as quorum-sensing inhibitors, could be…
A woman had the misfortune of losing her daughter to a rare disease caused by a reaction to medication. Now, she's speaking out to raise awareness for this debilitating condition.…
According to The Washington Post, on Tuesday the Food and Drug Administration, FDA, made history by approving a gene therapy for childhood blindness, which will change the lives of many…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
A recent study at Sanford Burnham Prebys Medical casts a ray of hope in treating multiple hereditary exostoses (MHE). MHE is a rare genetic disorder characterized by multiple noncancerous bone…
A 9-year-old boy, whose family has decided to remain anonymous, is finding relief after suffering from an incurable skin disease. According to his father, the boy was near death and…
Samuel Augustine was born 15 weeks early at Jackson Memorial Hospital with breathing problems and question marks surrounding his health. He was born with microcephaly and instantly placed on a…
Miracle workers at Queens University are working on a revolutionary method to treat patients with Lyme disease. Lyme disease is an infectious disease that springs from a tick bite's bacteria.…
Jay and Kathy Keller of Franklin, Idaho are getting creative this year with their donation efforts, reports WENY News. The Kellers are asking for people to write Christmas letters to…
Michele Holbrook is a runner-- if you spotted her jogging on the street, you wouldn't believe that her body was plagued by more than 20 tumors due to a rare…
Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
Gideon hides it well with his infectious smile, but for over two years, he fought bravely for his life. It started when he was a mere 6-year-old toddler. Gideon had…
Pharmaceutical company, Pfizer Inc., announced recently that the U.S. FDA approved the first (and only) Janus kinase inhibitor for treating severe psoriatic arthritis (PsA). Called XELJANZ® and XELJANZ® XR, this…
….Now what? A master’s degree in teaching? Would that even make sense for my life if I couldn’t even work an eight hour day and was currently seeking employment for…
Originally reported by Fox 17 Nashville, many Tennessee residents stress that a new tax reform bill will eliminate continued drug advancement and development for many rare diseases. The House and…
According to a December 9th press release from Janssen Biotech, Inc, the results of a pooled analysis of patients with mantle cell lymphoma illustrated that those treated with Imbruvica (ibrutinib) had…
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