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.

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

Exsilio Therapeutics Launches with $82 Million to Develop mRNA-Based Genomic Medicines Under Tal Zaks’ Leadership

Exsilio Therapeutics, mRNA, genomic medicines, Tal Zaks, gene therapy, gene editing, lipid nanoparticle delivery, redosing, safety profile, gene therapy approval

Argenx’s Vyvgart Hytrulo Secures Second US Approval for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Vyvgart Hytrulo, Argenx, FDA Approval, Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Autoimmune Disease, Peripheral Nervous System Disorder