Cambridge, UK – The saying “Knowing is half the battle” is never more true than when discussing early treatment of disease. Muscular dystrophy is one such disease where patients can benefit from early treatment. Now, new research is moving doctors and scientists closer to disease diagnosis in advance of patient...
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CAMBRIDGE, England – Researchers have discovered that misreading of therapeutic mRNAs by the cell’s decoding machinery can cause an unintended immune response in the body. They have identified the sequence within the mRNA that causes this to occur and found a way to prevent ‘off-target’ immune responses to enable the...
BALTIMORE, MD — Researchers at Kennedy Krieger Institute and Boston Children’s Hospital have discovered a new way to decrease early seizure likelihood and improve neurologic outcomes among infants with Sturge-Weber Syndrome (SWS). This collaboration, funded by National Institutes of Health, is the first multi-center study of its kind focused on...
Troy, New York – Using artificial intelligence tools to analyze years of biomedical data, researchers at Rensselaer Polytechnic Institute have discovered a possible connection between sleep, gastrointestinal health, and two potentially harmful behaviors often associated with profound autism: self-injury and aggression. Their study is published in the Journal of Personalized...
Leipzig, Germany – Every year, around 4,000 people in Germany are diagnosed with myelodysplastic neoplasms (MDS). The disease can lead to anaemia, infections and an increased risk of bleeding. People with higher-risk MDS have a significantly lower life expectancy than the average person of the same age. In the higher-risk...
MIAMI, Florida — Resolve Therapeutics, LLC, a mid-stage clinical development biotechnology company pioneering first-in-class therapies for autoimmune diseases, today announced the publication of results from its phase 2a clinical trial of RSLV-132 in patients with Systemic Lupus Erythematosus (SLE) in Lupus Science & Medicine. The publication entitled “Evaluation of RNase...
SOUTH PLAINFIELD, N.J., – PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented pooled placebo data from multiple studies in which the results confirmed the clinical benefit of EMFLAZA® (deflazacort) over prednisone for the treatment of Duchenne muscular dystrophy (DMD). The results demonstrated that DMD patients on daily EMFLAZA performed better on...
JINAN, China — The results of the phase II study on Qilu Pharmaceutical’s novel anticancer drug iparomlimab and tuvonralimab (QL1706) were published online in Signal Transduction and Targeted Therapy (Impact Factor=39.3). The study investigated the use of QL1706 in combination with chemotherapy, with or without bevacizumab, for the treatment of...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented a new analysis of five-year results that shows its novel gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, a previously intractable, fatal and devastating rare disorder of the central nervous system....
PETALUMA, Calif. — RetinalGeniX™ Technologies Inc. today announced that it has contracted with MEDsan, Inc. to provide diagnostic testing services for its Institutional Review Board (IRB) to conduct a study to personalize medical evaluations for patients receiving treatment for wet macular degeneration. “We are pleased to extend our services into...