The family of a four-year-old girl with a rare genetic disorder are looking for auction and raffle prizes to help give something back to two charities that have helped her. Bethany Hepburn, of Washwood Close, Little Hulton, has Wolf Hirschhorn Syndrome, which severely restricts her development and has caused a...
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Givinostat halted muscle shrinkage and fat infiltration in men with Becker muscular dystrophy (BMD) despite failing to ease muscle scarring, compared to a placebo, according to top-line, one-year results of an ongoing Phase 2 clinical trial. “We are very encouraged by the significant difference in muscle fat infiltration between the two groups after...
ALISO VIEJO, Calif. — Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders, and retinal diseases, announced today the U.S. Centers for Medicare and Medicaid Services (CMS) has assigned a unique, permanent Healthcare Common Procedure Coding System...
Primary and key secondary endpoints met, demonstrating a rapid, significant and sustained improvement in cholestatic pruritus and itch-related sleep interference versus placebo · Cholestatic pruritus presents in the majority of PBC patients, with debilitating impacts on quality of life including sleep disturbance · Late-breaking results presented at the European Association for the...
SEATTLE – According to Coherent Market Insights, the global myeloproliferative neoplasm drugs market is estimated to be valued at US$ 8,379.5 million in 2020 and is expected to exhibit a CAGR of 3.9% over the forecast period (2020-2027). Key Trends and Analysis: The increasing incidence of myeloproliferative neoplasm is expected...
Nerandomilast, an investigational agent, met the primary endpoint of both phase III trials, FIBRONEER™-IPF and FIBRONEER™-ILD, significantly reducing the decline in forced vital capacity (FVC) [mL] by absolute change from baseline at week 52, compared to placebo.1,2 Nerandomilast’s safety and tolerability profile was consistent across both trials, with similar rates...
SHANGHAI, China — At 15:00 Beijing Time (09:00 am Berlin Time) on October 20, the “Global Launch Event for Iza-bren (EGFR×HER3 Bispecific ADC) Nasopharyngeal Carcinoma BL-B01D1-303 Research Results” was held at the venue of the 2025 European Society for Medical Oncology (ESMO) Congress in Berlin. eChinaHealth held a forum on progress of nasopharyngeal carcinoma,...
AMSTERDAM, The Netherlands — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today new data showing that a one-time administration of its lead product GlyberaTM results in significant long-term health benefits. Long-term follow-up data from two clinical trials show that one administration with...
ROCKVILLE, Md. – GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians at University of California (UC) Davis Comprehensive Cancer Center initiated dosing of the first patient in a clinical study of uproleselan combined with venetoclax and azacitidine for the treatment of older or unfit patients with treatment-naïve acute myeloid leukemia...
SAN CARLOS, Calif.— Glycomine, Inc. announced today that enrollment has been completed in the Phase 2b POLAR study, a global, randomized, double-blind, placebo-controlled clinical trial evaluating GLM101 for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). PMM2-CDG is a rare genetic disorder that causes serious neurological and multisystem impairments....