Take Part in the Pompe Disease Registry
Patient registries are a great way for patients to get involved in shaping future research around their conditions. Many times, getting involved in a rare disease (or rare disorder)…
Patient registries are a great way for patients to get involved in shaping future research around their conditions. Many times, getting involved in a rare disease (or rare disorder)…
According to a story from The Intercept, Gilead Sciences' drug remdesivir is being touted in the media as a major development in the treatment of COVID-19, the illness caused by…
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…
Though many researchers explored topics of genetics and genomics over the years, the two fields are now emerging as key players within the realm of health and medicine. One…
According to a story from Sarcoidosis News, while the global challenge of the COVID-19 pandemic has been an obstacle for advancement in many rare diseases with trials and research grinding…
According to a recent article in Medscape, the FDA has issued accelerated approval for Bristol-Myers Squibb's Pomalyst (pomalidomide). Pomalyst was approved as the first new oral treatment for AIDS-related…
As reported in GSK, the FDA has just granted priority review to Nucala (mepolizumab), a targeted biological treatment to help patients with hypereosinophilic syndrome (HES). If the medication is accepted,…
As reported in QS Higher Education News, scientists at the Institute of Living Systems of the IKBFU and Kemerovo State University said they have improved a potential treatment option for PKU: the…
Ultomiris, a product of Alexion Pharmaceuticals, has recently been studied in a Phase 3 clinical trial. This study was intended to ultomiris' effect on life-threatening complications of atypical hemolytic uremic…
According to a story from Neurology Advisor, a recent study has found that patients living with neuromyelitis optica spectrum disorder (NMOSD) are at an increased risk of experiencing bone loss,…
Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…
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…
Lupus Nephritis Systemic lupus erythematosus (SLE) is a rare, chronic condition which affects a wide array of tissues in the body. A condition which signifies severe SLE progression is lupus…
In a recent article that appeared in Science Magazine, Amy Boland described her twelve-year quest to find a cure for the cancer that invaded her lymph system. At first,…
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 globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
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…
Many myasthenia gravis patients do not benefit from already existing therapies, such as steroids, acetylcholinesterase inhibitors, or immunosuppressants. It is this fact that has pushed many biotechnology companies to start…
As reported in Parkinson's News Today; the way that COVID-19 interacts with pre-existing conditions is now being uncovered as the novel virus makes it way across the world. A new…
Daily use of Nuplazid (pimavanserin) leads to strong and sustained reductions in the frequency and severity of hallucinations and delusions in people with Parkinson’s disease psychosis, according to data from…
Being a healthcare worker can be difficult: long hours, lots of moving parts, exposure to many patients and illnesses, and tons of policies and procedures to follow. But being a healthcare worker…
Unfortunately, many people with sickle cell disease are at an increased risk of experiencing central nervous system complications that can reduce quality of life. But more importantly, complications like stroke,…
In a recent article that appeared in Science Magazine, Amy Boland described her twelve-year quest to find a cure for the cancer that invaded her lymph system. At first,…
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…
When I was younger, I was diagnosed with Kawasaki disease, an inflammatory disease that impacts the coronary arteries. As I've grown up, I've become increasingly interested in how that…