Target ALS has recently launched a new research initiative, in which they will dedicate $15 million to amyotrophic lateral sclerosis (ALS) research, specifically the discovery of biomarkers. These millions will…
Author: Harsha K Rajasimha, MS, Ph.D. Editor: Sharlene Brown, Ph.D. When clinical investigators and study coordinators start planning a new study design, speed and efficiency are paramount. Running a clinical…
According to a story from Markets Insider, the biotechnology company REGENXBIO Inc. has recently released data from its phase 1/2 clinical trial. This is interim data from cohorts 1 and…
According to a story from Cancer Therapy Advisor, a recent phase 1 clinical trial investigated an experimental CAR T-cell therapy in heavily pretreated patients living with multiple myeloma, a rare…
Rare disease patients often face a long, complicated, and frustrating journey to the right diagnosis and treatment. Ally Trainor, a 21-year-old student at George Mason, is no exception. She has…
According to a story from blogs.biomedcentral.com, a research project called SCOPE H2020 focused on the impacts of chronic kidney disease screening of the elderly in the EU. The research included…
According to a story from Newswise, the early results of a recent phase 1/2 clinical trial have revealed the potential of an experimental treatment that was being tested for blastic…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…
A recent study sought to evaluate the impact of family history on the risk of autoimmune diseases, particularly primary biliary cholangitis and autoimmune hepatitis. There hasn't been much research conducted…
The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
The Yale Cancer Center has recently presented the findings of their study of the cost-effectiveness of caplacizumab at the 62nc ASH Annual Meeting and Exposition. Their conclusion, which is that…
The United States is coping with an obesity epidemic. It is therefore critical to understand the distribution of body fat and its effect on treatment. A recent article in Healio…
by Lauren Taylor from In The Cloud Copy Pantothenate kinase-associated neurodegeneration or PKAN is a rare, inherited condition in which the affected child experiences progressive degeneration in certain parts of…
According to a story in Charcot-Marie-Tooth News, the CMT&Me study has launched a competition in Europe with the purpose of raising awareness and money for CMT and related disorders. Any…
In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
A review of COVID-19 data from the UK shows that patients with rare autoimmune rheumatic disease (RAIRD) face a higher mortality risk, according to an article in Healio. The study,…
A study in the European Medical Journal aims to confirm the safety profile of vedolizumab, a treatment for inflammatory bowel disease (IBD). Specifically, they focused on acute infusion reactions. It…
According to a story from Hematology Advisor, a recent study released in Scientific Reports indicates that gene expression has the potential to predict both primary resistance and treatment response in patients living…
According to a story from GlobeNewswire, the biotechnology company Galecto, Inc., has recently published the results from its phase 2a clinical trial. This study was testing the company's experimental therapy…
While there is still a lot left to learn about COVID-19, there are a number of facts that we know, one of which is that children face less severe cases.…
Part of the National Institutes of Health (NIH), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and SteroTherapeutics have entered into a Cooperative Research and…
Transitions to adult care for patients who have a chronic respiratory disease, like cystic fibrosis, or a type of neuromuscular disease that influences the respiratory system is something that the medical…
A study published in Neurology has discovered a way to improve autoantibody testing in myasthenia gravis (MG) patients. An issue that physicians and patients face is false-positive results for neuromuscular or autoimmune…
The American College of Rheumatology presented new findings on November 5, 2020 at their virtual conference. They found that in patients with ANCA-associated vasculitis (AAV), hypothyroidism is associated with positive…
According to a story from ANCA Vasculitis News, a recent study has determined that monitoring changes in autoantibodies targeting the PR3 protein is useful for predicting ANCA vasculitis remission after…
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