A study recently published in the journal, Brain, has suggested that deficiencies in lysosomal storage genes may promote the development of Parkinson’s disease. Parkinson’s disease is a central nervous system…
When Avery was born, it didn't take long for her parents to realize that there was something different about her. She was not moving as much as most babies do,…
MonARC Bionetworks and the Pulmonary Fibrosis Foundation have partnered to bring the IPF community the PF Health app - a monumental partnership that will bring clinical research opportunities directly to…
Just like any other 7-year-old little boy, Ethan Perkins has big dreams. He loves trucks and reading, and wants to study fossils as a paleontologist reported KXLY. He also wants to be…
Hepatocellular carcinoma is a form of cancer that starts out in the liver. As far as cancers that begin in the liver, it's common, but it's still important to differentiate it…
Technology is hurting us. That's the claim made many times over the years. Smart phones may not have as many risks as some fear, but they can be a significant…
Pancreatic cancer is rare, and often, patients don't show symptoms until they're already in the later stages. Top pancreatic cancer centers in the country have been working together to find…
When she was only 16 months, Poppy was diagnosed with neurofibromatosis (NF). After 60 intense rounds of chemotherapy, she has now lost sight in one eye. NF is a rare…
While the development of orphan drugs for rare diseases has skyrocketed in the last 30 years, Dr. Scott Gottlieb, Commissioner of the Food and Drug Administration is recently questioning the…
Prader-Willi syndrome is a rare condition which causes the body to feel insatiable hunger, regardless of how much a patient eats. Treatment options are currently inadequate for many patients. Today,…
Lyme bacteria can survive a 28-day course of antibiotic treatment four months following infection by tick bite, according to a new study using a primate model for the disease. Despite…
The Spanish National Cancer Research Centre (CNIO) announced the discovery of a protein called RanBP6 as a regulator of epidural growth factor receptor (EGFR). The study, as originally printed in…
Originally reported by KVUE ABC, the race is on for the Rosenberg family to raise the funds that will benefit their 3-year-old child suffering from a rare brain disease known…
On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers…
The Canadian Fabry Association wants to create awareness for rare diseases and you can help! They want to get as many people to wear a Be Rare Be You tattoo…
Most people view Christmas as a time for celebrating. Spending time with family, and getting out to festive events and parties can be cornerstones of the holiday. For children like…
Growing up, my brother and I had a fun playhouse in our backyard with a huge slide and swings. We had the best times on that playhouse, playing games together…
It was a Christmas miracle for the Garcia family as their infant quickly recovered and was discharged from the hospital to enjoy the holiday at home, reported DailyMail. 1-year-old baby…
An Ottowa family is overjoyed after some good news. They have just been informed that their insurance company is offering to cover $120,000 worth of treatment to cover their son's…
Santa showed up early this year in a Chicago neighborhood, to the delight of dozens of children. However, his early visit to the area comes with an extra special purpose.…
Latest research from a University of Chicago team found that dynamic contrast-enhanced MRIs (DCE-MRIs) were much better in early detection of breast cancer than annual mammograms for patients with high-risk…
High school student, Molly Alter, fell limp while braiding her best friend, Jackie Acierno’s hair, and it was the heroic actions of Jackie to perform CPR that saved her life,…
Newborn baby Jada Elenbaas had to spend 287 days of her first year of life in the hospital. Jada was born with complete DiGeorge syndrome, also known as 22q11.2 deletion syndrome,…
Austin Brown was diagnosed with Wiskott-Aldrich syndrome at three months of age. An uncommon blood disease, Wiskott-Aldrich syndrome results in a weakened immune system. Thanks to a stem-cell bone marrow…
Caplacizumab is a medicine that has been developed to treat acquired thrombotic thrombocytopenic purpura (TTP). TTP causes serious symptoms. such as thrombocytopenia, a condition in which a patient doesn't have enough platelets. A…
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