We’re pleased to share a new publications in the Journal of Neuromuscular Diseases exploring 15 years of experience from the TREAT-NMD Advisory Committee for Therapeutics (TACT), highlighting how expert multidisciplinary review can help strengthen drug development programmes and improve chances of success for new therapies in neuromuscular diseases.
Since its launch in 2009, TACT has reviewed 90 therapeutic development programmes from both academia and industry, providing confidential advice from specialists across a wide range of disciplines, including preclinical research, clinical design, statistics, regulatory science, drug development and patient advocacy. The newly published review shares key lessons learned over this period, identifying common challenges that can delay progress or increase the risk of failure while offering practical insights to help developers navigate the complex path from laboratory research to clinical trials.
When TACT was first established,15 years ago, there was only one therapy that had been approved for neuromuscular disease. Today, multiple treatments are available and the development pipeline continues to expand. Highlighting how the field has evolved over the years.
Annemieke Aartsma-Rus, PhD, of Lieden University Medical Centre shares: “TACT was created to help de-risk therapeutic development programmes and increase the chance of effective therapies reaching the clinic. Fifteen years later, the continued demand for TACT reflects the value of bringing together both experienced expertise to support neuromuscular drug development.”
The review provides a unique perspective highlighting the challenges, lessons and opportunities shaping the future of neuromuscular therapy development and will be of great value to patients, patient organisations, researchers, clinicians, industry partners and anyone involved in bringing new treatments to the community.
Read the full article in the Journal of Neuromuscular Diseases.

