The Global Registry for Inherited Neuropathies was created by HNF to address the issue of the lack of quantitative and structured data collected on CMT disease. The registry collects private, de-identified data that is valuable for research, particularly for biotech and pharma. The registry helps researchers to gauge the effectiveness of CMT drugs by providing a large window of symptom data. The prevalence rates are important for CMT researchers to have a sufficient patient population for trials. Ultimately, the registry helps to advance our understanding of CMT and will help advance drug developments.
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.