Biophosphonates (BPs) are common treatments for osteoporosis and metastatic bone cancer. They help to prevent bone loss. Unfortunately, these drugs can have serious side effects. One of the most severe…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a story from finanzen.at, the biotechnology company Orbsen Therapeutics recently announced that its immunotherapy product ORBCEL-C is to be tested in a multi-site clinical trial called MERLIN that…
People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
According to a story from Medical Xpress, a recent study conducted by researchers from the UNC School of Medicine has illustrated that the accumulation of sticky, abnormal mucus, along with…
Mastocytosis Mastocytosis is a rare disease caused by mutations in the KIT gene. The condition causes mast cells to accumulate in the body. Depending on the location of these immune…
According to a publication from health website Stat, a new way of thinking about the price of drugs is taking root in Washington D.C. and beyond. "Value-based agreements" are a…
According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact…
Nigam Shah and Purvesh Khatri, professors at Stanford University, along with graduate student Madeleine Scott have just announced results from a research study which could be pivotal for those living…
Happy Spring! Anyone else got that spring fever? Unless you're getting tortured by allergies, it is a great time to be outdoors. The bees are buzzing, the flowers are blooming,…
Tracheostomies in ALS A tracheostomy is essentially an invasive procedure which helps patients breathe. It also removes secretions from their lungs. Doctors cut an opening into the patient's neck and…
According to a story from Radio NZ, two grandmothers have taken a central role in getting a petition delivered to the parliament of New Zealand. This petition is calling for…
Glioblastoma Glioblastoma is a rare cancer which begins in the brain. Approximately 20% of all tumors that occur in the brain are glioblastomas. These tumors are highly malignant because they…
According to a story from CheckOrphan, there are currently very few effective treatment options for chordoma, a rare, slow growing tumor that originates from bone. However, a recent research discovery…
RADOIR Rare Diseases Foundation of Iran (RADOIR) is an organization dedicated to supporting those living with rare diseases in Iran. It is a nonprofit which was founded in 2008 with…
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…
According to a story from MD Magazine, the team at Spatial Transcriptomics has utilized an innovative computational approach in order to map gene expression on the spinal cords of patients…
Bronchiectasis Bronchiectasis is a rare lung disease that currently has no cure and no effective treatment. It causes parts of the airways to become enlarged which results in lung damage.…
ACHM and XLRP Achromatopsia (ACHM) is a rare, inherited retinal disease which causes impaired cone photoreceptor function. Patients experience light sensitivity so extreme that it can cause blindness during the…
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An article in Austin Gastroenterology recently asks this question. It also confirmed what we already know, that genetic testing is now available to the average person and in fact,…
Optiz C Syndrome Optiz C syndrome (OCS) is an ultra-rare condition whose symptoms are often mistaken for Bohring-Optiz syndrome, Kabuki syndrome, or Kleefstra syndrome. The disease affects patients physical and…
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