Neurology

Sub Subramony, M.D., discusses the challenges faced by patients with Friedreich ataxia in accessing healthcare services and managing their condition, as well as the significant burden the disease places on families and caregivers.

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

Sub Subramony, M.D., discusses how the progression of patients' conditions often leads to a decline in quality of life and functional capabilities, highlighting key clinical indicators that necessitate the adoption of mobility aids.

Sub Subramony, M.D., discusses how the modified Friedreich Ataxia Rating Scale (mFARS) is valuable for monitoring disease progression in clinical trials and its applicability in everyday clinical practice for assessing patient status.

Panelists discuss how the differential diagnosis for Friedreich's Ataxia involves distinguishing it from other ataxias, the process of definitive diagnosis through genetic testing, and the significant emotional and practical impact that such a diagnosis has on affected families.

A Friedreich ataxia specialist discusses how the typical clinical presentation of Friedreich's Ataxia includes early onset with symptoms such as ataxia and sensory loss, while also examining the pathophysiology and incidence rate of the condition.

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.

A Friedreich ataxia specialist shares their optimistic outlook on future developments in the management of the condition.

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).

Looking to the future of Alzheimer disease treatment, the panel discusses key takeaways on the evolving therapeutic landscape.

Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.