According to a story from Healio, a recent study suggests that the addition of four more genes to the conventional genetic test used to diagnosed familial hypercholesterolemia could allow more…
Orencia Orencia (abatacept) is a treatment for juvenile idiopathic arthritis (JIA) that was approved for use in the U.S. for the pediatric patient population in 2008. In Japan, the therapy…
According to a story from MD Magazine, a recent study that was presented at Digestive Disease Week 2019 suggests that patients with ulcerative colitis, a rare form of inflammatory bowel…
According to a story from BioPortfolio, dogs have been making the lives of human beings better for thousands of years in many different ways, and now there is new one…
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Google’s Parent Company (Alphabet) Funds New Gene Editing Company An article recently published in MIT Review reports that Verve Therapeutics announced that it has received funding from Alphabet’s GV venture fund plus…
According to a story from the Daily Bruin, a team of scientists from UCLA are in the midst of developing the first therapy for multiple sclerosis that could address the…
According to a story from Global Genes, the drug developer Viela Bio recently issued a presentation of the results from a clinical trial that tested the company's investigational product candidate…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
According to a story from Global Genes, the drug developer Viela Bio recently issued a presentation of the results from a clinical trial that tested the company's investigational product candidate…
PREFER PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University's Welcome Centre for Mitochondrial Research in effort…
According to a story from Pulmonary Hypertension News, a recent study found that patients with disease-associated pulmonary arterial hypertension had similar rates of survival compared to patients with idiopathic pulmonary…
Phage Therapy Phages, or bacteriophages, are the natural enemy of viruses. They are the most abundant organisms in the biosphere. Researchers have used phages as treatments before, however after the…
According to a publication from the State University of New York at Buffalo, most symptoms of proximal renal tubular acidosis do not stem from the acidic blood levels that characterize…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…
According to a publication from ANCA Vasculitis News, a new study in International Kidney Reports suggests that stopping treatment of the disease once it goes into remission may lead to…
According to a story from Genome Web, a team of researchers from across the country have concluded an in-depth genetic analysis focused on the origins of Hirschsprung's disease, a rare…
The FDA has just recently approved CYRAMZA as a treatment for those with hepatocellular carcinoma (HCC), who have been treated with sorafenib, and who have ≥400 ng/mL of alpha-fetoprotein (AFP).…
According to a story from mirror.co.uk, Professor Selim Cellek is leading a clinical trial that will test a drug combination designed to treat erectile dysfunction associated with Peyronie's disease, a…
According to a story from apnews.com, the drug developer Ayala Pharmaceuticals has recently announced that the company's investigational product candidate, known as as AL101, has earned Orphan Drug Designation. This…
A story from findmecure.com describes several of the latest developments in the area of myasthenia gravis treatment that patients should be aware of. The field of rare disease treatment is…
According to a story from CityScene, Ohio State University's Dr. Michael Racke is considered one of the country's cutting edge researchers in the field of multiple sclerosis treatment. He is…
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Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta. According to a recent article in NPR News, this theory was…
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