The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
Gut bacteria has been known to play a role in a number of conditions, such as inflammatory bowel disease, rheumatoid arthritis, and more. A new study has discovered a connection…
We all know that diagnosing cancer earlier means better outcomes for patients. It’s also essential to monitor the spread of cancer in already diagnosed patients. If we can watch as…
HAE Junior is a Czech-based patient organization aiming to improve the quality of life of children & youth with hereditary angioedema (HAE). It was set up in December 2019 in…
Prevail Therapeutics, a company that focuses on developing gene therapies for neurodegenerative diseases, has announced that a composition of matter patent has been issued for PR001. This therapy is intended…
Sorreto Therapeutics recently announced through Globe Newswire that it has filed an application for its investigational new drug COVI-DROPS (STI-2099). The study will investigate the safety and efficacy of the…
Maintaining lung function is a major goal of cystic fibrosis (CF) treatment. In order to accomplish this goal, doctors will use airway clearance techniques, such as vest therapy. The problem…
This week Hallmark Movies and Mysteries premiered The Christmas Bow, which features Joy Perry, a woman with Charcot-Marie-Tooth disease, playing a woman with CMT. And while CMT has been known…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
According to an announcement from the FH Foundation, data from the FH Foundation’s CASCADE FH Registry, published in The Journal of Pediatrics, has determined that children living with familial hypercholesterolemia regularly…
According Newswise, in a new study first published in Gastroenterology, researchers at Cedars-Sinai have identified a set of genes, made up of rare genetic deviations and more common genetic patterns,…
As reported in Medical Xpress; Fight for Sight, a charity in the UK dedicated to vision, has an exciting announcement for people with macular diseases: they have just granted funding…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
According to a story from Newswise, a collaborative research team from the University of California San Diego School of Medicine and the Rady Children's Institute for Genomic Medicine will be…
Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…
According to an article from Biospace, Takeda Pharmaceuticals has released the results from their Phase 3 HELP trial, an extension study of TAKHZYRO. This hereditary angioedema (HAE) treatment has shown…
PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…
A recent analysis has shown that eosinophilic gastritis and eosinophilic duodenitis are severely undiagnosed or misdiagnosed as another GI condition. Allakos Inc. has recently reported the results of this study,…
At the Tufts University School of Medicine, researchers have uncovered a molecular mechanism that eventually leads to a build-up of amyloid beta, which is a major cause of Alzheimer's disease.…
Lung infections are a common symptom of cystic fibrosis, with Staphylococcus aureus being the most prevalent. While physicians can treat this infection using antibiotics, they do not yet understand its role…
Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…
Research on myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS), has developed in recent years; however, until now there has still been a gap in the literature regarding loneliness. This condition…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
According to Biospace, Acceleron Pharma has presented preliminary data from their ongoing Phase 2 trial of sotatercept, a treatment for pulmonary arterial hypertension (PAH). The study, titled SPECTRA, is designed…
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