A new case study suggests that the blood disorder, thalassemia, may be present in people with Gaucher disease. Both diagnosis and treatment become more complicated if this hypothesis proves true.…
Human beings have always speculated about the future. Science fiction is probably one of the most prominent examples, and stories are replete with robots, lasers and flying cars. But no…
According to an article at publicnow.com, the FDA has granted Breakthrough Therapy Designation to avelumab combined with INLYTA (axitinib) for people with advanced renal cell carcinoma. This designation should accelerate…
Good news for Prometic Life Science new drug application! The pharmaceutical company has been searching for a cure for idiopathic pulmonary fibrosis (IPF), a rare lung disease which you can…
Despite the obvious cliché nature of the old adage, knowledge really is power. Technology and medical advancements give doctors and researchers more information than anyone could have fathomed even a…
One of the medical research field's biggest developments recently - within the last 5 years! - is a new form of gene editing, also know as genome editing. Gene editing refers to…
Drug research is taking a plunge to the underwater abyss. The nation's opioid crisis has been declared a national public health emergency. Along with the Department of Defense, the University…
Good news for patients who suffer from ankylosing spondylitis (AS). There may soon be a new option for treating their disease if the results of a new trial support the…
A new study comparing two different treatments demonstrates that both could be effective in treating hydrocephalus. This condition is characterized by a buildup of cerebrospinal fluid inside the brain. In…
The more scientists learn about gut bacteria, the more fascinating it becomes. It can affect your mood, the foods you crave, and according two new studies, it can even change…
Sickle cell disease causes red blood cells to form abnormally, which disrupts oxygen function. Cancer Prevention Pharmaceuticals have been working with Vanderbilt Medical Center and NCI to start a new Phase…
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…
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…
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…
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…
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,…
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…
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,…
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