Boston, Mass. – An international, phase 3 clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare disease that can cause debilitating symptoms. The study, published in the New England Journal of Medicine, found that treatment with inebilizumab greatly reduced the symptoms of immunoglobulin...
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Burton-on-Trent, England – Clinigen, the global pharmaceutical services company, today launched a new campaign called ‘What is Possible?’ to help empower patients and medical professionals to ask what more is possible in the treatment of rare diseases. There are over 7,000 rare diseases impacting over 300 million people globally, and...
CHENGDU, China — Clover Biopharmaceuticals, Ltd., a global commercial-stage biotechnology company, today announced positive preliminary safety, efficacy and pharmacokinetics data in a Phase Ⅰ clinical trial evaluating SCB-219M, an innovative thrombopoietin receptor agonist (TPO-RA) mimetic bispecific Fc-fusion protein produced from CHO cells, for the treatment of cancer patients with chemotherapy-induced...
St. Louis, MO – Alzheimer’s disease has plagued one large Colombian family for generations, striking down half of its members in the prime of life. But one member of that family evaded what had seemed would be fate: Despite inheriting the genetic defect that caused her relatives to develop dementia...
Madrid, Spain – A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has made a breakthrough discovery in the understanding of cardiac arrhythmias by unraveling the complexities of Andersen-Tawil syndrome (ATS), an extremely rare inherited cardiac disorder. Led by Dr. José Jalife, head of the CNIC Cardiac Arrhythmia Group,...
Madrid, Spain – Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare genetic disease that affects just 1 in every 20 million people; it is estimated that fewer than 400 children in the world have the disease. HGPS is characterized by accelerated aging, severe atherosclerosis, and premature death at an average...
Madrid, Spain – Immunotherapy is already improving treatment options for many cancer types, but research groups keep exploring ways to boost the body’s immune response against the tumor. Researchers at the Spanish National Cancer Research Center (CNIO) and the 12 de Octubre University Hospital in Madrid have developed a new...
Madrid, Spain – Researchers at the Spanish National Cancer Research Centre (CNIO) propose a new treatment for brain metastases that respond poorly, or not at all, to immunotherapy, and provide a biomarker to predict in which cases it should be used. Our bodies have a mechanism to destroy anything that...
HOUSTON, TX – CNS Pharmaceuticals, Inc., a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, today announced completion of planned enrollment in its global potentially pivotal study evaluating Berubicin, the Company’s novel anthracycline and the first anthracycline...
Paris, France – Coave Therapeutics, a genetic medicine company focused on developing life-changing therapies, is delighted to announce that it is one of six organisations sharing $2.9 million in grants from the ALS Association that support the advancement of novel therapies for the treatment of amyotrophic lateral sclerosis (ALS). The...