Study Describes New Rare Disease: TRAF7 Syndrome
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
In a story from Multiple Sclerosis News Today, multiple sclerosis is known for its variability- one patient's experience may look nothing like a friend's with the same disease. For this…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
A team of researchers from the Perelman School of Medicine and several other institutions recently reported a significant reduction in tumor growth in myeloid immune cells using an experimental…
According to a story from UNC Health Care, the Belgian drug company Argenx recently announced that its investigational treatment efgartigimod has performed well in a phase 3 clinical trial. The…
A team of researchers from the Perelman School of Medicine and several other institutions recently reported a significant reduction in tumor growth in myeloid immune cells using an experimental…
In the past, genetics were hypothesized to be a potential cause of glaucoma and eye disease. Now, British researchers definitively linked SPATA13 gene mutations with a form of glaucoma called primary…
Because of COVID-19, many health and medical-related conferences and events have been moved to virtual settings. But that doesn't mean that the learning has stopped, says Healio. During the Heart…
Immune-Onc Therapeutics recently received clearance from the FDA to begin trials of IO-202, a treatment for acute myeloid leukemia (AML). This news comes after the cancer immunotherapy company filed an…
According to a story from Parkinson's News Today, an analysis of results from several different clinical trials suggests that the drug istradefylline (marketed as Nourianz) can reduce the duration of…
Medical professionals are already aware that inhaling air pollution can increase one's risk of a number of conditions, including respiratory and heart diseases. They are now discovering that it can…
According to MIT News, scientists recently discovered that inhibiting levels of the GSK3 alpha enzyme could reduce, or even reverse, symptoms of fragile X syndrome. Their study, which utilized mouse…
In 2015 families in Wales and England were given the good news that tolvaptan (JINARC®), which was developed for autosomal dominant polycystic kidney disease (ADPKD), will be available within one…
If you think about it, our brains are pretty amazing. It is one of our most complex features and contains tens of billions of neurons which help control the…
According to a story from Parkinson's News Today, the Vercise system, developed by Boston Scientific as a method of deep brain stimulation, was able to provide benefit to Parkinson's disease…
According to a story from Medical Xpress, an Italian study has found that people in urban areas are at a 29 percent greater risk of developing multiple sclerosis (MS). This…
According to a story from Parkinson's News Today, ropinirole (marketed as Requip) skin patches proved capable of improving motor function in patients with advanced Parkinson's disease when compared to placebo.…
The virtually-held National Kidney Foundation 2020 Spring Clinical Meetings explored various topics relating to nephrological conditions, treatment, and patient outcomes. One presentation, spearheaded by Dr. Adam Weinstein, MD, and…
In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…
As reported in Guru Focus, biotech company Protagonist Therapeutics has announced progression in their Phase 2 study on PTG-300 as treatment for patients with polycythemia vera, a rare cancer. Dr.…
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
Women can do anything that men can do, and vice versa. Well, while that sentiment may be true in our everyday life, it isn't quite true on a genetic…
According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…
Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
By Danielle Bradshaw from In The Cloud Copy Researchers in Germany have gone on record saying that there is a concern with how well patients are able to tolerate the…