According to a story from the National Gaucher Foundation, a significant challenge for many patients with Gaucher disease are the effects that the illness can have on bones. Common treatments…
According to a story from news-medical.net, the process that causes the neurons responsible for voluntary muscle movement in amyotrophic lateral sclerosis (also known as Lou Gehrig's disease) to die is…
LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…
Alnylam Pharmaceuticals has been developing lumasiran as a potential treatment for primary hyperoxaluria type 1 (PH1). This investigation began with ILLUMINATE-A, a clinical trial whose results should be announced by…
According to a story from EurekAlert!, a new mathematical approach pioneered by a team of scientists from New York University and Cincinnati Children's could greatly improve the analysis of scientific…
Personalized medicine is the latest craze in patient care. In essence, it refers to the development of treatment options which take into account the patient as an individual. It reduces…
For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF).…
According to a press release from American biotechnology company Intercept Pharmaceuticals, the company recently released additional data supporting its phase 3 clinical trial of obeticholic acid (OCA) in the treatment…
According to a story from Rocket News, the class of drugs known for their "gene silencing" mechanism of action appear to be effective in treating porphyria, a rare disease that…
It's one thing to cope with a new rare disease diagnosis. It's another entirely to cope with that diagnosis and then four years later learn that diagnosis is incorrect and…
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 press release from Inovio Pharmaceuticals, the American biotech company's experimental recurrent respiratory papillomatosis (RRP) drug INO-3106 showed promising results at the end of a pilot clinical study…
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 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 Neuroscience News, a team of researchers has managed to examine how amyotrophic lateral sclerosis (ALS) progresses on a cellular level for the first time. The…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
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An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…
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…
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…
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…
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