Cambridge, England – An international team has shown that the injection of a type of stem cell into the brains of patients living with progressive multiple sclerosis (MS) is safe, well tolerated and has a long-lasting effect that appears to protect the brain from further damage. The study, led by...
treatment News
Philadelphia – Thanksgiving is often a time for thinking about your belly. For those with an inflammatory bowel diseases like ulcerative colitis, feasting can be associated with stress, even when food isn’t a trigger for the painful symptoms. New research in mice suggests that certain foods – especially those high...
BOULDER, Colo. — Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne). Sevasemten is an investigational...
BOULDER, Colo. — Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of Duchenne. EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage...
BEIJING & CAMBRIDGE, Mass. – EdiGene, Inc. (“Company”, or “EdiGene”), today announced the first patient enrolled in multicenter Phase I clinical study of its investigational gene-editing hematopoietic stem cell therapy ET-01 for patients with transfusion dependent β-thalassemia at the Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, and...
London, England – A drug candidate, based on pioneering UCL and Moorfields Eye Hospital research and currently under development by SIFI S.p.A., has been found to be highly effective in treating a rare sight-threatening eye infection in a new international clinical trial. The findings, published in Ophthalmology, describe the efficacy and...
Treatment with monoclonal antibodies (mAbs) and anticytokine biologics is not effective in decreasing the frequency of coronary artery aneurysms (CAAs) in patients with Kawasaki disease (KD), but the incidence of treatment resistance may be reduced with these agents compared with conventional intravenous immunoglobulin (IVIG) therapy alone, according to findings from...
FREIENBACH, Switzerland – EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Bronchitol® (inhaled mannitol) for the treatment of cystic fibrosis (CF) in adults and in children aged 6 years and above as add‐on to other medicines. Bronchitol® is...
Stockholm, Sweden — Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) announced that the Company, as part of its initiatives to increase disease awareness about MCT8 deficiency, has participated and informed about MCT8 deficiency, at the following medical conferences during the first quarter of 2023: British Paediatric Neurology Association (BPNA) Annual...
PALO ALTO, Calif. — Eiger BioPharmaceuticals, Inc, a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, today announced that it and its partner AnGes, Inc. received marketing approval from the Ministry of Health, Labour and Welfare for Zokinvy (lonafarnib), a treatment for Hutchinson-Gilford progeria...