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Lausanne, Switzerland – Neurodegenerative diseases, such as Alzheimer’s and Parkinson’s, present a significant health challenge, affecting over 50 million people globally. One common feature of these diseases is the accumulation of misfolded protein aggregates in the brain, known as amyloid fibrils, which disrupt normal cell function and eventually lead to...
UXBRIDGE, England — Norgine, a leading European specialty pharmaceutical company, is pleased to announce that Swissmedic has approved the registration of IFINWIL® (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB)1. “This milestone marks a further regulatory approval of IFINWIL® for high-risk neuroblastoma, following approvals in the US, Israel and Australia,” said Jörg Plessl,...
A large clinical trial has found that postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer that has spread to as many as three lymph nodes and who have a 21-gene recurrence score (RS) of 25 or lower can safely skip having chemotherapy and...
By Raffaele Pereno – PhD, MBA AMSTERDAM, the Netherlands – SynaptixBio, a UK-based biopharmaceutical company pioneering antisense oligonucleotide (ASO) therapies for ultra-rare neurological diseases, presented its groundbreaking work on TUBB4A-related leukodystrophy (also known as H-ABC) at the World Orphan Drug Congress (WODC) in Amsterdam. CheckOrphan spoke with SynaptixBio’s CEO, Dan...
WALTHAM, Mass. — Syndax Pharmaceuticals Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with relapsed/refractory (R/R) mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth...
WALTHAM, Mass. and WILMINGTON, Del. – Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) and Incyte (Nasdaq: INCY) announced today that they have entered into an exclusive worldwide collaboration and license agreement to develop and commercialize axatilimab, Syndax’s anti-CSF-1R monoclonal antibody. “This partnership has the potential to significantly expand and maximize the axatilimab...
CAMBRIDGE, Mass. – Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced that it has initiated the Phase 1 clinical trial of SYNB1934, an investigational Synthetic Biotic™ medicine for the treatment of Phenylketonuria (PKU). “We are delighted to have so quickly advanced...