Nusinersen for the treatment of spinal muscular atrophy will get a follow-up.
spinal muscular atrophy (SMA)
Posted inMetabolism, Neuroscience, Oncology, Pharma & Biotech, Rare/Orphan Diseases
Alltrna: World’s First Drugs Based on Transfer RNAs
A completely new therapeutic modality will help treat both rare and common diseases.
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review
Novartis revealed the most expensive drug in the world. And it is amazing.
