Aarhus, Denmark – NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, announces today a poster and late-breaking oral presentation describing safety and efficacy data from its Phase 2a SYNAPSE-CMT study evaluating ignaseclant (formerly known as NMD670) in patients living with Charcot-Marie-Tooth disease...
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Curing cancer has been the dream of countless doctors and researchers over the decades. Yet some types of cancer, whether because they affect relatively few people or lack influential advocates, receive less attention and funding than others. A recent gift to Harvard Medical School (HMS) is boosting efforts to understand...
An experimental stem-cell treatment developed by StemCells of Palo Alto has shown no dangerous side effects after being injected into six children with a rare and as-yet always fatal brain disorder, the company said Monday. The groundbreaking study begun in 2006 involves children suffering from Batten disease, a heretofore incurable...
Nivolumab with or without ipilimumab as maintenance therapy after first-line chemotherapy failed to prolong survival among patients with extensive-disease small cell lung cancer (ED-SCLC), according to the results of the CheckMate 451 trial published in the Journal of Clinical Oncology. Although most patients with ED-SCLC respond to first-line chemotherapy, recurrence is...
BOSTON, Mass. – NodThera, a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases, today announces positive data from its Phase Ib/IIa study in Parkinson’s disease patients, evaluating the effects of its oral, brain-penetrant NLRP3 inflammasome inhibitor NT-0796, on inflammatory and disease-specific biomarkers in the blood...
Deep vein thrombosis (DVT) after laparoscopic hepatectomy may be high, and no effective risk assessment methods exist. The authors of a study published in the Journal of Hepatocellular Carcinoma estimated incidence of DVT after surgery and created a DVT risk nomogram. The western hemisphere has well-established guidelines for thromboprophylaxis, but its application...
WASHINGTON, DC — The future of rare disease advancements will take center stage at the National Organization for Rare Disorders (NORD®) Rare Disease and Orphan Products Breakthrough Summit®, Oct. 19-21 in Washington. NORD is proud to announce the top researchers selected for the Lightning Rounds Poster Presentations session. Chosen from a competitive...
AMSTERDAM, Netherlands and LOUISVILLE, Ky. – NORGINE B.V. (Norgine) a leading European specialist pharmaceutical company and US WorldMeds (USWM), a Kentucky-based specialty pharmaceutical company, today announced an exclusive licensing agreement by which Norgine will register and commercialise difluoromethylornithine, DFMO (eflornithine) in Europe, Commonwealth of Independent States, Australia and New Zealand. DFMO is a repurposed molecule investigated for use...
AMSTERDAM, the Netherlands — Norgine, a leading European specialty pharmaceutical company, today announced that the European Commission (EC) has granted marketing authorisation for mavorixafor as XOLREMDI® following a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).1 The marketing authorisation was granted under exceptional circumstances*, reflecting the ultra-rare nature...
AMSTERDAM, the Netherlands — Norgine B.V., a leading European specialty pharmaceutical company, today welcomes the positive opinion issued by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending the granting of a marketing authorisation under exceptional circumstances[*] for XOLREMDI® (mavorixafor) in patients aged 12 years and older for...