The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis ...
QALSODY is the first therapy designed to address the underlying genetic cause of SOD1-ALS2, a rapidly progressive and uniformly fatal condition that impacts motor function, speech, and respiratory ...
HTA assessments highlight importance of timely and equitable access to QALSODY for this ultra-rare genetic form of ALS and urgent need for therapies that slow progression, preserve function and ...
Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being ...
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