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Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi  announced positive pivotal Phase 3 results from a trial evaluating Dupixent® (dupilumab) in adults with uncontrolled prurigo nodularis, a chronic type 2 inflammatory skin disease that causes extreme itch and skin lesions. The trial met its primary and all key secondary endpoints, showing...
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for DYN101, an...
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, today announced its recognition of Rare Disease Day 2021. Dynacure is helping to raise awareness of myotubular and...
WALTHAM, Mass. — Dyne Therapeutics, Inc., a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced positive initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its DELIVER trial of DYNE-251...
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...
Dystonia sounds more like an Eastern European country than a health disorder. And like so many other diseases, conditions and disorders, the average person has probably never heard of it. It is only when dystonia affects a friend or family member that people start to ask questions. What is dystonia?...
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...