Friedreich Ataxia: Looking Forward at the Treatment Landscape

A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).

In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

The HERCULES study of tolebrutinib is the first and only to show reduced confirmed disability progression at 6 months in nonrelapsing secondary progressive multiple sclerosis (MS).

Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior balance.

Study results demonstrated significant correlations between fatigue and reductions in walking speed and mobility among patients with multiple sclerosis.

Engaging with healthy lifestyle behaviors, including a healthy diet and physical activity, was associated with better outcomes among patients with multiple sclerosis (MS).