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...
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SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C. — United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that The Lancet has published full results of its ADVANCE OUTCOMES study, available here. Ralinepag has not been approved for use in any indication by the FDA and remains investigational for PAH. Ralinepag reduced the risk...
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 — George Dahlman, senior vice president of public policy for The Leukemia & Lymphoma Society (LLS), testified today before the U.S. Senate Subcommittee on Defense Appropriations, calling for funding for a dedicated, stand-alone blood cancer research program at the Department of Defense (DoD). The Institute of Medicine (IOM) has...
PRINCETON, N.J. – The McQuade Center for Strategic Research and Development, LLC (MSRD), a member of the global Otsuka family of pharmaceutical companies, announces today that it has entered an agreement with Sentinel Oncology (Sentinel), a drug discovery company. The McQuade Center for Strategic Research and Development at Otsuka in...
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...
Amsterdam, The Netherlands – A new stool test appears to detect colorectal cancer precursors better than the current test. This could further reduce the number of new colorectal cancer cases as well as the number of people dying from the disease. A study led by the Netherlands Cancer Institute compared...
SAN DIEGO, Calif. — Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the final results from the completed Phase 1/2 MARINA® trial of delpacibart etedesiran (del-desiran) in people living with myotonic dystrophy type 1...