Clinical trial diversity is not a new concept–the U.S. Food and Drug Administration (FDA) issued a draft guidance providing specific recommendations to industry on how to improve diversity in clinical trials in April 2022 but the passage of the Food and Drug Omnibus Reform Act, or FDORA, highlighted that the...
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Santa Barbara, Calif. — It’s official: The ketogenic diet proved to be effective at controlling polycystic kidney disease (PKD) in the first randomized controlled clinical trial of ketogenic metabolic therapy for PKD. “I’m really happy about these clinical trial results,” said UC Santa Barbara biologist Thomas Weimbs, whose lab was...
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...
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 – Scientists at the Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), working in collaboration with the Instituto de Investigación Biomédica de Barcelona (IRB Barcelona), have discovered a new immunotherapy strategy that reduces cancer recurrence in mouse experimental models. The study, published in Nature Communications, shows that a specific...
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 – Cutaneous melanoma, the most aggressive form of skin cancer, is characterised by its accumulation of a large number of mutations. Although some of these alterations should be recognised as a threat by our defences, melanomas often escape immune system surveillance. As a result, more than half of...