QUT researchers have signed an agreement with US pharmaceutical company Quoin to fund a pre-clinical program at QUT to further research a potential treatment for a rare skin condition called Netherton Syndrome. Research will focus on Netherton Syndrome (NS), an hereditary autoimmune condition NS is characterised by skin inflammation, itchiness,...
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RARE-X, a collaborative platform for patient-controlled data collection, global data sharing and analysis, is partnering with the Broad Institute of MIT and Harvard to advance and support the discovery of new treatments for 9,500 rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS). The collaboration will leverage Broad Institute’s technology to assist in patient data collection...
Montreal, Canada – In Quebec, it is estimated that more than 500,000 people suffer from a rare disease, which is defined as a disease that affects less than one in every 2,000 citizens. Too often, affected patients and their families are left in the dark when it comes to finding...
BOSTON, Mass. — Ratio Therapeutics Inc. (Ratio), a pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, announced today that dosing of the first cohort has been completed in the ATLAS trial, a Phase 1/2 open-label study evaluating the safety, tolerability, and efficacy of the company’s lead...
BERKELEY, Calif. — Ray Therapeutics (RayTx), an industry-leading clinical-stage biopharmaceutical company pioneering differentiated vision restoration therapies for those living with severe retinal degenerations, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to its lead program, RTx-015, for the treatment of retinitis pigmentosa (RP). PRIME...
BERKELEY, Calif. — Ray Therapeutics (RayTx), a clinical-stage biopharmaceutical company pioneering optogenetic therapies for those living with retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead candidate, RTx-015, for the treatment of retinitis pigmentosa (RP)....
Dublin, Ireland – Researchers in the School of Population Health at RCSI University of Medicine and Health Sciences have provided new evidence of the health benefits of weight loss efforts that lead to diabetes remission for type 2 diabetes patients. For participants in the weight-loss trial who were able to...
Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
Dublin, Ireland – A new partnership between RCSI University of Medicine and Health Sciences and Irish diagnostics company Serosep Ltd is set to personalise treatment for ulcerative colitis and ultimately improve quality of life for patients with the condition. Under the new alliance, RCSI researchers will work with Serosep to...
LA JOLLA, CA — Nerve cells in the brain demand an enormous amount of energy to survive and maintain their connections for communicating with other nerve cells. In Alzheimer’s disease, the ability to make energy is compromised, and the connections between nerve cells (called synapses) eventually come apart and wither,...