Platform trials for DMD..

This looks interesting and the first public meeting was chaired by PPMD in September 2019..

@waynesworld or anyone else have knowledge of this platform trial situation??

I assume ANP’s trials would fit the bill. Background: Duchenne muscular dystrophy has a deep drug development pipeline, with increasing numbers of interventions being developed, and over 30 active clinical trials ongoing. Among neuromuscular diseases, only ALS has more interventions being developed. However, drug development has been slow, given the lack of transformational therapeutics that show efficacy with short duration treatment, the heterogeneity of the disease, the small number of eligible patients for current trials, concern regarding randomization to placebo, and endpoints that are only responsive to treatment effects in certain stages of the disease. Trials therefore have prolonged enrolment periods and longer times to complete as participants wait for read-outs.

https://www.rehabpub.com/conditions/neurological/muscular-dystrophy-conditions/duchenne-stakeholders-meeting-sept-9-discuss-platform-trial/

https://www.mdaconference.org/node/926

https://www.ctap-duchenne.org/platform/