3 results
Patients with ALS are routinely prescribed riluzole, the only registered drug for the treatment of ALS. This study will evaluate the concentration-effect relationship of riluzole (100 mg) and retigabine (300 mg) on axonal excitability measures. In…
The primary objective is:• To evaluate the safety and tolerability of a single local i.c. dose of SI-053 to establish the MTD and/or the RP2D (dose escalation) and to further evaluate safety at the RP2D (dose expansion).The secondary objective is:•…
The purpose of this first-in-human study is to evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses (MADs) of DYNE-101 administered intravenously (IV) to participants with DM1.…