Researchers have recently conducted a study using mouse models of Charcot-Marie-Tooth (CMT) disease, titled "CMTM6 Expressed on the Adaxonal Scwann Cells Surface Restricts Axonal Diameters in Peripheral Nerves." It was…
Waking up to the news has one encouraging note, another new day and another new drug is being tested. According to an article in Biospace, the news from Sorrento Therapeutics…
As published in a press release by the Hereditary Neuropathy Foundation, a project funded by the National Institutes of Health (NIH), researchers at the University of Rochester, the University of Pennsylvania, and…
When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
According to a recent report in the Journal of Clinical Oncology, over one thousand patients with high-risk stage III melanoma were included in the Keynote-054 clinical trial (NCT02362594). The…
According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…
A research team from IPM.ai, a subsidiary of Swoop, and Insmed, a biotechnology company, recently collaborated on a case study that demonstrated how AI and machine learning could be used…
PRNewswire recently carried BERG’s announcement of the results of its COVID-19 clinical study. BERG, a biotech company based in Framingham, Mass., utilizes artificial intelligence in its research of diseases and…
Eisai has announced that they will begin a Phase 3 trial, titled MOMENTUM, to evaluate lorcaserin as a treatment for Dravet syndrome. This decision comes after a consultation with the…
PracticeUpdate recently published an article relating to a study with the primary goal of lightening the burden that many males experience regarding long-term prostate surveillance. Active surveillance has been preferred…
A study reported in NewsWise evaluates retinoid-x receptor (RXR) agonists as a treatment for recurring and refractory multiple sclerosis (MS). This specific RXR agonist, bexarotene, was found to help remyelination,…
According to an article from BioSpace, the TargetCancer Foundation has began enrolling patients in their TRACK study. This study is intended to provide rare cancer patients with individualized treatment options…
Dr. Jasvinder Singh, a rheumatologist at Alabama’s University in Birmingham is the author of a study that was featured in Arthritis Care and Research. During a recent interview with MedPageToday,…
Damian Green from the University of Washington is the Principal Investigator for a new research project in multiple myeloma. This project is thanks to a research agreement between the Fred…
Dr. Lisa Sammaritano from Weill Cornell Medical College has explained a way to lower risks within pregnancy for those with rheumatic diseases. Pregnancy and Rheumatic Diseases Patients who have a…
APT-1011 is a treatment for eosinophilic esophagitis, a condition that currently faces an unmet medical need. The medication was originally developed by TPG Capital, but they created a new company,…
According to an article in Diagnostic Imaging, MRIs and PET scans show that Parkinson’s disease may start as either body-first or brain-first, in effect behaving like two different diseases.…
Target RWE, an innovative health evidence solutions company based in Durham, North Carolina recently announced its current data from the study of nonalcoholic steatohepatitis (NASH) via PRNewswire. The study, entitled…
by Danielle Bradshaw from In The Cloud Copy There was a case report (called Anti-MUSK positive myasthenia gravis with antiLrp-4 and antigen antibodies) released recently that talked about a patient…
Medical professionals have already discovered a number of genes that play a role in pulmonary arterial hypertension (PAH), but according to LabRoots, they have found a new one. The ABCC8…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
A research team at Ghent University in collaboration with Imec recently released preliminary data on their artificial iris. They are calling it a "smart contact lens." By embedding microelectronics into…
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