English (United Kingdom)

https://curated-unify.zendy.io/wp-json/zendy-region/v1/featured_content/oa?rat=en

https://curated-unify.zendy.io/wp-json/zendy-region/v1/highlighted_journal/

Global: Zendy Plus annual

Presents the access of premium content as premium feature

Premium Content

Presents the keyphrase highlighting as premium feature

Keyphrase Highlighting

Presents the summarisation as premium feature

Summarisation

Insights

Presents the pdf analysis as premium feature

PDF Analysis

Presents the zaia usage as premium feature

ZAIA

Global: Zendy Tools annual

Global: Zendy Free Plan

Global: Zendy Tools monthly

Global: Zendy Plus monthly

Purpose of Review People with fatal neuromuscular diseases such as ALS want to access investigational products. Trials are our preferred pathway for this, but most people with these diseases will not be able to participate due to restrictive inclusion criteria, travel burdens, or design features they will not accept. This leaves FDA Expanded Access Programs (EAPs), the Right To Try (RTT) pathway, and self-purchase of alternative and off-label treatments (AOTs). Recent Findings A recent survey highlighted physician barriers to the above pathways, including lack of knowledge and concerns about time burdens and risks. Emerging resources are highlighted that can mitigate some of these concerns. Summary With the information in this chapter, we hope that neuromuscular clinicians will feel more knowledgeable and confident in supporting patient request for investigational products.

Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers