Disease or Syndrome
Soleno breaks through with FDA nod for Prader-Willi syndrome treatment Vykat XR
FDA approval, Vykat XR, Prader-Willi syndrome, hyperphagia, Soleno Therapeutics, diazoxide choline
Wave Life Sciences’ WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Treatment
Duchenne muscular dystrophy (DMD), WVE-N531, exon 53 skipping, Phase II trial, dystrophin expression, muscle health improvement, FDA approval
Epicrispr raises $68M, will start clinical trial of epigenetic editing for FSHD
Muscular Dystrophy, Facioscapulohumeral, Epicrispr, Clinical Trials, EPI-321
FDA Approves Blujepa: First New Oral Antibiotic for Uncomplicated UTIs in Nearly 30 Years
Gepotidacin, Blujepa, GSK, uncomplicated urinary tract infections (uUTIs), FDA approval, first-in-class antibiotic
Eyeing precision medicines for ocular diseases, Character clinches $93M
Glycogen storage disease type II, Personality Character
Merck Acquires Rights to Hengrui’s Oral Lp(a) Inhibitor in $2B Deal
Merck, Hengrui Pharmaceuticals, HRS-5346, Lp(a) inhibitor, cardiovascular disease, atherosclerosis, licensing agreement
Hillstar Bio Launches with $67M to Develop Precision Immunotherapies for Autoimmune Diseases
Precision immunology, autoimmune diseases, TRBV9+ T cells, axial spondyloarthritis, Series A funding
Merck Secures $2B Deal with China’s Hengrui for Novel Heart Drug
Merck, Hengrui Pharmaceuticals, HRS-5346, Lipoprotein(a) inhibitor, cardiovascular disease, licensing agreement
GSK Launches Groundbreaking Study on Shingrix and Dementia Risk Reduction
GSK, Shingrix, dementia, UK Dementia Research Institute, Health Data Research UK, NHS data, real-world experiment
Surrozen sets its sights on ophthalmology after binning liver programme
Ophthalmology specialty, Severe (severity modifier)