Neuromuscular

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Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases

Published by NIH

NeuromuscularClinical Trial Readiness

This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical studies that address critical needs for clinical trial readiness in rare neurological and neuromuscular diseases. These studies should result in clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials. Through the support of trial readiness studies, NINDS expects to enhance the quality and increase the likelihood of success of clinical trials in these rare diseases.

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Advisory Committee for Therapeutics (ACT) Toolkit

Published by Newcastle University

Research and Translational ResourceNetworkExpert AdviceClinical TrialNeuromuscular

The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary model centred upon an international group of academic and industry drug development experts, including patient representatives. TACT gives advice on the translational and development pathway of therapeutic programs in rare Neuromuscular Diseases. Due to its success in the neuromuscular field, a toolkit to replicate the TACT model has been produced. The Advisory Committee for Therapeutics (ACT) toolkit is intended to support rare disease communities outside of Neuromuscular to establish their own ACT, by providing them with step-by-step guidance and template documentation.

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