Emerging Science & Pipeline

In recognition of International Ataxia Awareness Day, observed annually on September 25, NeurologyLive® reviews 6 investigational agents in clinical development across ataxia-related disorders.

Seven experimental DM1 therapies show promise and pitfalls; see latest trial data, a major phase 3 miss, and key 2026 readouts.

Emerging therapies for muscular dystrophy are targeting a range of disease mechanisms, from RNA-based approaches and muscle-directed delivery to cell therapy and DUX4 suppression.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive® reviews the investigational agents currently moving through clinical development for spinal muscular atrophy.

A look at where things stand for Fragile X syndrome drug development, covering seven agents across four mechanisms as the field regroups from two recent Phase 3 setbacks.