Amsterdam, Netherlands – The drug vamorolone (Agamree®) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has recently been approved for clinical use in the United States by the Food and Drug Administration (FDA) and in the EU by the European Medicines Agency (EMA)....
treatment News
Rockville, MD – According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapies hold the greatest potential to transform the Gaucher disease landscape. While the importance of these novel therapies has been emphasised, there remains a significant amount of groundwork to be covered before these therapies receive...
NEW YORK, NY — EyeDura Therapeutics Inc., a leader in preservative-free, topical sustained-release ophthalmic therapies, today announced initiating preclinical efficacy studies with the Singapore Eye Research Institute (SERI) towards continued development of its lead insulin-based topical eye drop treatment for neurotrophic keratitis (NK), a rare eye disease affecting approximately 71,000...
WATERTOWN, Mass.— EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a company committed to developing and commercializing therapeutics to improve the lives of patients with serious retinal diseases, today announced results from new subgroup analyses from the Phase 2 DAVIO 2 clinical trial of EYP-1901, an investigational sustained delivery maintenance treatment for wet...
Washington, DC – URAC, the nation’s pre-eminent pharmacy accrediting body, announced that Fairview Pharmacy Services, part of Fairview Health Services, received Rare Disease Pharmacy Designation. The pharmacy is among the very early adopters of this designation which shows their commitment to high-quality pharmacy practice and advanced expertise with rare disease...
LIBERTYVILLE, Ill. — Families of Spinal Muscular Atrophy has just released details of the latest developments for the Families of SMA funded therapeutic pipeline. Families of SMA is currently funding three distinct drug discovery programs: 1) Quinazolines to boost gene expression; 2) Tetracyclines at Paratek Pharmaceuticals to correct gene splicing;...
Basel, Switzerland – Fampridine is currently used to improve walking ability in multiple sclerosis. A new study shows that it could also help individuals with reduced working memory, as seen in mental health conditions like schizophrenia or depression. Remembering a code for long enough to type it in; holding a...
TURKU, Finland and BOSTON, Mass. — Faron Pharmaceuticals Ltd., a clinical-stage biopharmaceutical company pursuing a CLEVER approach to reprogramming myeloid cells to activate anti-tumor immunity in hematological and solid tumor microenvironments, today announced that the first patient has been dosed in Phase 2 of the BEXMAB trial that evaluates the...
TURKU, FINLAND – Faron Pharmaceuticals Ltd. (AIM:FARN, First North:FARON), a clinical-stage biopharmaceutical company, today announced updated full data from its Phase 1/2 BEXMAB study, presented at the American Society of Hematology (ASH) 2025 Annual Meeting (oral and poster presentation). The data highlights significant improvement in survival outcome and new findings on...
TURKU, Finland – Faron Pharmaceuticals Ltd. (HEL:FARON)(LSE:FARN), a clinical-stage biopharmaceutical company focused on developing novel treatments for medical conditions with significant unmet needs, today announced that two abstracts featuring data from the Phase 1/2 BEXMAB study have been accepted for presentation at the American Society of Hematology (ASH) 2025 Annual Meeting....