CMT&Me is an observational, non-interventional study, sponsored by Pharnext, to collect Real-World Evidence (RWE) from people living with CMT. The objective of the study is to better understand the impact of the disease on patients’ daily lives: the burden of CMT, its natural history and treatment, and medical, social and pharmacoeconomic effects.
Breaking News for SORD Deficiency Patients
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.
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