NMD Pharma initiates Phase 2 study of NMD670 in patients with Charcot-Marie-Tooth disease type 1 and 2
- NMD Pharma has started a Phase 2a clinical trial for NMD670, targeting Charcot-Marie-Tooth (CMT) disease types 1 and 2.
- The trial, named SYNAPSE-CMT, is a randomized, double-blind, placebo-controlled study involving 80 adult patients.
- Key endpoints include changes in the 6-minute walk test, 10-meter walk/run test, and timed-up-and-go test over a 21-day period.
- The study is being conducted across clinical sites in the US and Europe, with new sites announced until March 2025.
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Intellia announces first clinical evidence from phase 1 study of nexiguran ziclumeran in ATTR amyloidosis
- Intellia's Phase 1 trial of nexiguran ziclumeran (nex-z) shows promising results in ATTR amyloidosis, with significant TTR reduction and disease stabilization.
- The trial includes patients with ATTR-CM and ATTRv-PN, showing stability or improvement in cardiac and neuropathy markers after a single dose.
- Nex-z demonstrated a favorable safety profile, with mild to moderate infusion-related reactions as the most common adverse events.
- Intellia is advancing to Phase 3 studies, MAGNITUDE and MAGNITUDE-2, to further evaluate the efficacy and safety of nex-z.
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Neurogene updates on NGN-401 gene therapy trial for Rett syndrome
- Neurogene reports a serious adverse event (SAE) in a participant of the NGN-401 Phase 1/2 trial for Rett syndrome.
- The SAE involved a systemic hyperinflammatory syndrome in a high-dose cohort participant.
- The FDA has allowed the trial to continue with a lower dose of 1E15 vg, pausing the high-dose cohort.
- No other treatment-related SAEs have been reported in the trial.
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