A recent Phase 4 study has shown that Opsumit can improve function of the right heart ventricle for patients with pulmonary arterial hypertension (PAH). The right ventricle pumps blood to…
From social distancing and shuttered businesses to personal health concerns, COVID-19 has changed daily life for the foreseeable future. There are nearly 4.75 million diagnoses worldwide, with 1.53 million in…
Multiple biotechnology and pharmaceutical companies have joined in the fight against COVID-19. But one, Gilead Sciences, has been at the forefront of both progress and controversy. The company developed remdesivir,…
A recent article in US News and World Report gives an account of the first patient to have reprogrammed cells successfully implanted in his brain. The patient’s doctors reported that…
In her early 20's, Mackenzie Alleman lost her mother to breast cancer. Then, at 28, she herself underwent a preventative double mastectomy after learning that she carried the mutated BRCA1 gene.…
In January 2020, doctors flocked to the ACR Winter Symposium to discuss new trends, treatments, and burgeoning research within the field of rheumatology. Dr. Jeffrey Curtis, MD, MS, MPH presented…
Recently, scientists from Emory University set out to discover whether there was any pathological link between spinocerebellar ataxia type 36 (SCA36) and amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). Both…
A recent article in Parkinson’s News Today described new findings associated with mutations in the LRRK2 gene, which are associated with Parkinson’s disease (PD). Indications are that patients with…
Biological robots, commonly known as biobots, mimic the actions of other organisms (e.g. jumping or swimming). Biobots are composed of artificial materials and also organic material. As published in…
Atypical hemolytic uremic syndrome (aHUS) is a rare condition which can be sparked by pregnancy, although it can also occur without it. Essentially, if you are genetically predisposed to the condition,…
We need controls! No, not cockpit controls or videogame controls, and no, we're not out of control. We need control participants for the Dupuytren Biomarker Discovery Pilot Study. Our goal is…
The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…
A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…
As reported in Medical Xpress, the U.S. Centers for Disease Control and Prevention have listed antibiotic resistance as one of the most pressing health challenges of our time. Infection treatment…
A recent BioSpace press release shares some exciting news for patients with non-small cell lung cancer, medullary thyroid cancer, and other thyroid cancers caused by RET gene mutations. Retevmo (selpercatinib) is the…
Ali Hardy struggled with her health for a long time. In an article for The Mighty, Ali, an actress in New York City, explains that she was never able to…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…
In 2003, Suzan Jackson was diagnosed with myalgic encephalitis, or chronic fatigue syndrome (ME/CFS). Shortly after, she was faced with the complexity of having a chronic illness. Should she tell…
The Change in Hospitals Due to COVID-19 Between 10 and 15 percent of all hospital staff is forced to isolate due to the virus at any one time. This means…
Happy Friday! If you're observing Memorial Day, enjoy the upcoming long weekend. Going to use the time to brush up on a little rare disease news? We've got three articles…
Contact Tracing Contract Tracers are an essential component to stopping the spread of COVID-19. Essentially, it entails finding those exposed to the virus and quarantining them until they are no…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
Scientists say some myelin-damaging disorders have a distinctive pathology that groups them into a unique disease entity. The white knob on the left shows demyelination of a nerve fiber end.…
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