FIREFISH 2-year data show continued benefit of risdiplam for type 1 SMA

The 24-month results from part 2 of the open-label FIREFISH trial suggest that prolonged treatment with risdiplam is associated with continued improvements in motor function and development in infants with type 1 spinal muscular atrophy.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Research identifies sex differences in SSc-ILD outcomes

Men with systemic sclerosis-associated interstitial lung disease may have a more severe disease course than women, whereas women with the condition could be more likely to experience certain adverse events when treated with immunosuppressive therapy, suggest findings from two studies.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

VALOR hints at potential benefit of tofersen for SOD1 amyotrophic lateral sclerosis

Data from the open-label extension of the VALOR trial suggest that treatment with the intrathecally administered antisense oligonucleotide tofersen may modify the disease course of amyotrophic lateral sclerosis due to SOD1 mutations and lead to improvements in function.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.