Waltham, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping, was granted U.S. Food and Drug...
treatment News
WOODCLIFF LAKE, N.J. – Eagle Pharmaceuticals, Inc. (“Eagle” or the “Company”) (NASDAQ: EGRX) today announced that TREAKISYM ready-to-dilute (“RTD”) (bendamustine hydrochloride 120 mg/m2) liquid formulation has been approved for a new indication in combination with rituximab (“BR therapy”) as treatment for relapsed or refractory diffuse large B-cell lymphoma (“r/r DLBCL”)...
RESEARCH TRIANGLE PARK, N.C. – BioCryst Pharmaceuticals, Inc. today announced that the French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d’Utilisation de cohorte (cohort ATU), or Temporary Authorization for Use, for the use of berotralstat to prevent attacks of hereditary angioedema (HAE) in...
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...
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...