The Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual registration. 

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Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice

Published by Neuromuscular Disorders

TREAT-NMDMiceNeuromuscular diseases

The authors propose the introduction of a standard study design for the mdx mouse model. Several aspects, including animal care, sampling times and choice of tissues, as well as recommended endpoints and methodologies are addressed and, for each aspect, a standard procedure is proposed. Testing of all new molecules/drugs using a widely accepted and agreed upon standard experimental protocol would greatly improve the power of pre-clinical experimentations and help identifying promising therapies for the translation into clinical trials for boys with Duchenne Muscular Dystrophy

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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

Published by The Company of Biologists Ltd

TREAT-NMDNeuromuscular diseasesMouse models

This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases. It emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.

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