Manchester, UK – The first patient in the UK has been treated with a pioneering new investigational gene therapy designed to treat the debilitating genetic disorder Gaucher disease. Experts at Northern Care Alliance NHS Foundation Trust (NCA) say the research study is a major milestone for the inherited condition. They...
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SAN FRANCISCO – Perspectum announced recruitment of the first patient in a multi-center collaborative study to characterize Primary Sclerosing Cholangitis in children, “Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children.” This 10-year collaborative study includes 12 geographically-diverse children’s hospitals in the United States and one Canadian children’s hospital....
FORT LEE, N.J. – Kedrion Biopharma, an international biopharmaceutical company specialized in the manufacture and distribution of plasma-derived therapeutic products used in treating rare and serious diseases, announced that the first patient has been treated in the KIDCARES10 (KIg10 in peDiatric subjects: effiCacy, sAfety and phaRmacokinEticS of a 10% IG) clinical trial. The...
Eddie Ndopu, an award-winning disability activist from South Africa, and one of 17 United Nations advocates for the Sustainable Development Goals, lives with spinal muscular atrophy, and faces many difficulty daily challenges. Ahead of International Day of Persons with Disabilities, on 3 December, Mr. Ndopu discusses how he has overcome...
MILAN, Italy – Newron Pharmaceuticals S.p.A. (“Newron”), a research and development company focused on novel CNS and pain therapies, announced today that detailed results of the first phase III clinical trial of safinamide as adjunctive treatment in mid- to late-stage Parkinson’s disease (PD) (Study 016), conducted with its partner Merck...
West Des Moines, Iowa – Born in 2013, identical twins Kenzie and Kaylie Morell were hitting some, if not all, of their normal infant and toddler milestones—until they began to slip back from them. When Kenzie was around 15 months old, she started to regress. Kaylie followed a year and...
BEIJING & CAMBRIDGE, Mass. – CANbridge Pharmaceuticals, Inc., a biopharmaceutical company developing and commercializing innovative drugs to treat rare diseases and targeted cancers in China and globally, announced that the first subject has been dosed in the Phase I trial of CAN106 in Singapore, which is being developed for the...
Lund – Active Biotech (NASDAQ STOCKHOLM: ACTI) today announced that the first subject has been dosed in phase I clinical study of the newly developed eye-drop formulation of the company’s candidate drug laquinimod. Laquinimod is being developed as a new treatment for non-infectious non-anterior uveitis and has the potential to...
BOCA RATON, Fla. — First Wave BioPharma, Inc., a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that it has entered a non-binding term sheet to sell its Niclosamide program for the treatment of inflammatory bowel diseases (IBD) such as ulcerative...
The Gamida Cell-Teva Joint Venture announced today that five prestigious cord blood transplantations centers in Spain, three in Barcelona and two in Valencia, have joined the ExCell study. Participating clinical sites include, in alphabetical order: * Hospital Clínic of Barcelona, Barcelona: Principal Investigator, Dr. Enric Carreras, MD * Hospital Clínico...