Wave Life Sciences Achieves Breakthrough in Huntington’s Disease Treatment with WVE-003, Eyes Accelerated Approval

Wave Life Sciences, Huntington’s disease, WVE-003, Phase Ib/IIa study, accelerated approval, mutant huntingtin protein, allele-selective silencing, RNA medicine

Sarepta Therapeutics: Next Steps After Full FDA Approval for Duchenne Muscular Dystrophy Gene Therapy

Sarepta Therapeutics, Duchenne muscular dystrophy, gene therapy, FDA approval, Elevidys, microdystrophin, ambulatory patients, non-ambulatory patients, pipeline development, rare diseases, genetic medicine.