According to a story on PRNewswire, the biopharmaceutical company, Enzyvant, kicked off their Farber Disease Natural History Study in October. Acid ceramidase deficiency, also known as Farber disease, is an obscure…
An international study conducted by researchers at the University of Copenhagen has discovered an essential mechanism that controls wound healing in the intestine. The new discovery shows that the cells…
Doctors from the Colorado University School of Medicine recently conducted research indicating that an exercise protein could help prevent the onset of Parkinson’s disease symptoms. Parkinson’s disease is a rare…
An engineering science professor from British Columbia designed an all new high resolution scanner that will enable eye problems to be diagnosed earlier. This will also allow earlier treatment and…
Orla Tinsley, an Irish-born journalist living in New York, has had to put her work in hold and is now on life support at New York-Presbyterian Hospital. As a sufferer…
A couple of days before Christmas, the FDA granted a regular approval to the drug, hydroxyurea (Siklos) for treatment of pediatric sickle cell anemia. Sickle cell anemia is a chronic…
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…
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…
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…
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…
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…
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,…
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…
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…
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