Nothing is worse for a family than hearing the word “cancer,” especially when it involves a child. The Tyson family was ready to start their perfect life eleven years ago.…
When you are going through the daily struggles of living with a rare disease, you feel alone. It is hard to accept the support from family and friends because they…
I am always incredibly inspired by the people that don’t allow their rare diseases to prevent them from doing what they love. Whether it’s been Charity Tillemann-Dick with opera or Olivia…
Have you been told that you had to stop doing your passion in life because of your rare disease? This is the story of opera lover, Charity Tillemann-Dick. Her life…
Olivia Ports recently performed for over a thousand people, including the Mayor and Governor of Chicago, helping to raise $3.75 million for research by Lurie Children’s Hospital in only one…
A new study from researchers at UCSF found that a simple tool could optimize kidney treatment in children, by reducing dialysis and increasing pre-emptive kidney transplants. The tool is called…
Ionis and Roche have licensed together for the development of IONIS-HTTRx, a breakthrough drug in the treatment of Huntington’s disease. Huntington’s disease is a rare neurological condition that attacks the…
The National Organization for Rare Disorders (NORD) is the largest nonprofit independent organization for rare disease information, research, and advocacy. The organization is devoted to the treatment of all rare…
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…
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…
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…
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…
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…
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…
Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…
Researchers from MIT have recently identified a new way to stop the growth of glioblastoma brain tumors, bringing renewed hope in treatment options for this rare life-threatening cancer. Glioblastoma is…
Vertex Pharmaceuticals announced in late August that the U.S. FDA accepted applications of tezacaftor/ivacaftor for patients with cystic fibrosis (CF). Specifically, this combination treatment can be used in patients ages…
Natalie Stokes was always the curvy one in her group of friends. But when she started putting on more and more weight despite cutting down to only 1,000 calories a…
This year marks the end of a second decade of achievements for EURORDIS, now formally known as EURORDIS-Rare Diseases Europe. EURORDIS is a non-governmental alliance that is the voice of…
Most people do not enjoy going to the dentist, but for patients with down syndrome, this normal task is even more of an ordeal. Patients often have a difficult time…
An easier diagnosis may be coming for ulcerative colitis patients, as researchers at Georgia State University recently used a special type of spectroscopy to examine the blood serum of mice…
While there is good scientific evidence backing up the fact that omega-3 fatty acids found in fish oil can help slow or even prevent heart disease, but does fish oil…
As autoimmune disorders become an increasing problem throughout the world, particularly in developed countries, there has been an increasing amount of research dedicated to finding a cure, and the role…
In breaking news for the progeria community, biomedical engineers have just succeeded in growing miniature human blood vessels that mimic progeria, the ultra-rare genetic condition characterized by premature aging in…
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