Neurology

Patisiran for hereditary transthyretin amyloidosis polyneuropathy in V122I and T60A genotypes is reported with variant-specific outcomes. Findings describe measurable changes in neurologic function and patient-reported domains under routine multicenter cond...

In hereditary transthyretin amyloidosis with polyneuropathy, the siRNA therapy patisiran reduces hepatic TTR production, aiming to slow axonal injury. In individuals with V122I or T60A variants, results indicate neuropathy can stabilize or improve on treatm...

Amyotrophic lateral sclerosis imposes profound fatigue and mobility limits that shape willingness to join clinical research . Interviews illuminated how energy conservation, travel burden, invasive procedures, and altruism interact. Key finding: participant...

In amyotrophic lateral sclerosis, participation hinges on the mechanism of energy conservation versus participant burden. Key finding: individuals favor short, low-burden procedures , flexible scheduling, remote and home-based options, and transparent recip...

Amyotrophic lateral sclerosis limits energy and mobility, making traditional trial operations taxing. Participants value altruism but resist avoidable burden. The key finding: reduce travel, compress or replace in-person visits, and enable remote or at-home...

Patisiran for hereditary transthyretin amyloidosis polyneuropathy with V122I or T60A variants was evaluated in a phase IV multicenter setting, reporting genotype-specific outcomes under routine practice. Results described changes in neuropathy severity and ...