EAST LANSING, Michigan – Barth syndrome (BTHS) is an ultra-rare but debilitating genetic disease affecting cell mitochondria. High infant mortality due to cardiac complications and a weakened immune system significantly decrease life expectancy. Current medications treat symptoms but not the cause, leaving an unmet clinical need to develop effective treatments...
treatment News
Tokyo, Japan – Leukemia is a common term used to refer to a form of blood cancer. However, there are different types of leukemia depending on the cell type involved. One unique form is myeloid/natural killer (NK) cell precursor acute leukemia (MNKPL). Because of its rarity, there is no consensus...
Bellingham, Washington – In the realm of biomedical sciences, the quest for accurate and efficient diagnostic tools is ever-evolving. One such promising innovation making waves is the photoacoustic (PA) technique. In the past decade, PA imaging has emerged as a viable imaging modality demonstrated in many clinical applications with promising...
SEOUL, South Korea and HYDERABAD, India — The International Vaccine Institute (IVI), an international organization with a mission to discover, develop, and deliver safe, effective, and affordable vaccines for global health, today announced that it has commenced a technology transfer of simplified Oral Cholera Vaccine (OCV-S) to Biological E. Limited...
PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of...
Relacorilant plus nab-paclitaxel improves progression-free and overall survival in patients with platinum-resistant ovarian cancer, with no need for biomarker selection Addition of relacorilant did not increase side-effects, compared to nab-paclitaxel monotherapy Results presented in late-breaking podium presentation at ASCO 2025 with simultaneous publication in The Lancet REDWOOD CITY, Calif. — Corcept...
Marks the First-Ever Global Pediatric Trial for Menin Inhibitors The PedAL (Pediatric Acute Leukemia) Master Trial is at the heart of LLS’s Dare to Dream Project to transform treatment and care for kids with blood cancer WASHINGTON, DC — The Leukemia & Lymphoma Society (LLS) announced today the first...
WASHINGTON, DC — The Melanoma Research Alliance (MRA), the largest non-profit funder of melanoma research worldwide, welcomes the U.S. Food and Drug Administration (FDA) decision to approve Iovance Biotherapeutics’ AMTAGVI™ (lifileucel) for the treatment of patients with advanced melanoma in the second line treatment setting. AMTAGVI is the first individualized...
STOCKHOLM, Sweden and WALTHAM, Mass, USA – Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that The New England Journal of Medicine (NEJM) published results from the phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving...
NEWARK, Calif. — CymaBay Therapeutics Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that The New England Journal of Medicine (NEJM) has published detailed results from the RESPONSE Phase 3 trial evaluating seladelpar, an investigational agent, and the...