DURHAM, N.C. – Though exceedingly rare, some people diagnosed with amyotrophic lateral sclerosis (ALS) partially or fully recover from the lethal neurodegenerative disease. A better understanding of this baffling phenomenon, reported in medical literature for at least 60 years, could point to potential new treatment approaches. To that end, researchers...
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DURHAM, N.C. – Biomedical engineers at Duke University have developed a new technique to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B (LGMD2B). The approach grows complex, functional 3D muscle tissue from stem cells in the laboratory,...
Durham, North Carolina – Scientists from Duke-NUS Medical School (Duke-NUS) have developed a new approach using the Zika virus to destroy brain cancer cells and inhibit tumor growth, while sparing healthy cells. Using Zika virus vaccine candidates developed at Duke-NUS, the team discovered how these strains target rapidly proliferating cells...
Approval based on pivotal results showing improvements in sustained disease remission and reductions in itch and oral corticosteroid use compared to placebo in adults with BP BP is a chronic, debilitating, and relapsing rare skin disease affecting approximately 27,000 adults in the US whose disease is uncontrolled by systemic corticosteroids...
TARRYTOWN, N.Y. and PARIS, France — Regeneron Pharmaceuticals, Inc. and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of pediatric patients aged 1 to 11 years, weighing at least 15 kg, with eosinophilic esophagitis (EoE). Dupixent is now the first...
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi announced positive pivotal Phase 3 results from a trial evaluating Dupixent® (dupilumab) in adults with uncontrolled prurigo nodularis, a chronic type 2 inflammatory skin disease that causes extreme itch and skin lesions. The trial met its primary and all key secondary endpoints, showing...
Abstract Background: Advanced systemic mastocytosis (AdvSM) is a rare, life-threatening myeloid neoplasm predominantly driven by KIT D816V mutations. Avapritinib is a selective and potent KIT D816V inhibitor approved for the treatment of AdvSM. Methods: PATHFINDER (NCT03580655) is a clinical study evaluating avapritinib in adults with AdvSM. This analysis reports long-term...
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for DYN101, an...
STRASBOURG, France and PHILADELPHIA, Jan. 26, 2021 – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the U.S. Patent and Trademark Office (USPTO) has granted U.S. Patent No....
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, today announced its recognition of Rare Disease Day 2021. Dynacure is helping to raise awareness of myotubular and...