Nikki J
Moderator
- Joined
- Mar 22, 2012
- Messages
- 16,384
- Reason
- PALS
- Diagnosis
- 04/2014
- Country
- US
- State
- MA
- City
- Boston
What this means is it is approved but if ATLAS the carrier prevention trial fails the fda could rescind approval. However this is a great moment even though tofersen only benefits a small number of PALS. It is the culmination of many years of research and trials of two different sod1 asos ( the first was promising but needed tweaking. )
FDA approves treatment of ALS associated with a mutation in the supero
FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based o
www.fda.gov