It's been two years since the Zika outbreak and now, two-year-olds born with microcephaly due to the infernal outbreak, are still suffering through long-term problems. Children who developed health problems…
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…
NORD, the National Organization for Rare Diseases, sent out their happiest wishes for the holidays, their utmost gratitude for 2017, as well as their plan of attack for the New…
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…
After over a decade, Debbie Zelman, founder of Debbie's Dream Foundation, succumbed to her stomach cancer at age fifty. From the beginning, Debbie's prognosis was not good. When the cancer…
According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…
On Christmas Day, Chris Bombardier, a man suffering from hemophilia B, traveled to Antarctica. While most of the people that go there scientists, Chris has a completely different goal in…
Originally reported by The Straits Times, the Chew family from Singapore does everything they can to keep their nine-year-old daughter alive, even if it means spending their life savings and…
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…
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