source
- FDA approves Ionis’ Zanvastro as first disease-modifying therapy for Alexander diseaseThe U.S. Food and Drug Administration has granted approval to Ionis Pharmaceuticals' antisense oligonucleotide Zanvastro, the first therapy that modifies disease progression for Alexander disease.…
- Ultragenyx to consider major cost cuts after Angelman drug fails trialUltragenyx announced that its experimental therapy GTX-102 (apazunersen) for Angelman syndrome failed to meet primary and secondary endpoints in a Phase 3 study. The company said there were no dif…