Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide...
12 KB (860 words) - 04:18, 7 September 2024
for familial ALS, particularly as there is now a licensed gene therapy (tofersen) specifically targeted to carriers of SOD-1 ALS. A shortage of genetic...
131 KB (14,038 words) - 11:12, 29 September 2024
greatly enhances delivery efficiency and cell-type specific targeting. Tofersen (marketed as Qalsody) was approved by the FDA for the treatment of SOD1-...
23 KB (2,219 words) - 02:24, 20 August 2024
pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx...
16 KB (1,428 words) - 12:29, 20 September 2024
Sodium phenylbutyrate and ursodoxicoltaurine N07XX21 Eplontersen N07XX22 Tofersen N07XX23 Troriluzole N07XX59 Dextromethorphan, combinations "ATC (Anatomical...
2 KB (282 words) - 23:28, 21 December 2023