CMD Clinical Trial Readiness Campaign. The CMD genes ~ The CMD to LGMD spectrum. Congenital Myopathy- RYR1. There is an overlap between congenital muscular dystrophy and congenital myopathy .
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Being ready means:1. We maximize our chances of getting a good drug to and through clinical trial.2. We are able to measure an effect that the FDA accepts and is meaningful to our community.
The drug has no effect, not the intended effect or the size of the effect is not measurable.
The community was not “ready” to go to clinical trials.
How do we measure functional gains in strength?
Current motor scales and timed tests
Effort dependent, how do the scales work in the setting of contractures
A scientific investment that delivers a drug to slow disease progression, buys time, and improves quality of life
Earlier diagnosis with genetic confirmation (if possible)
Less variable, more proactive medical care from the global medical community with an emphasis on multidisciplinary care