Thanks gassy appreciate your input but i prefer this version I...

Thanks gassy appreciate your input

but i prefer this version

I wasnt suggesting that MRI be used as a surrate endpoint to replace pul2 but it has been accepted as a secondary endpoint This is a fact as i stated Duvyzat used it in their trial in their Ambulant boys

With reference to the difficulty in getting into the Mri and wheel chair access etc etc

In our Phase 2 trial did we not include MRI in that trial

were these boys Ambulant or none ambulant i believe they were none ambulant

We had 9 none ambulant boys in our 2A trial and 9 none ambulant boys managed to go through the MRI scanning process

Is this not a contradictory statement above or is it just another excuse for lack of funding

Taken from that link i gave you to read

The UF team is currently contributing MR measurements to a number of clinical trials, including gene therapy studies and additional studies of Duvyzat in patients who have lost the ability to walk. The team also leads a study of the natural progression of Becker muscular dystrophy.

Duvyzat are going through a trial with none ambulant boys see below

The ULYSSES trial is a Phase 3 clinical trial that will test Duvyzat (givinostat) in boys with Duchenne muscular dystrophy (DMD) who are no longer able to walk

The ULYSSES trial will enroll up to 138 boys with DMD.

Taken from the duvyzat results

Additional data was considered for its ability to serve as confirmatory evidence. The percentage of fat fraction present in the vastus lateralis (VL) muscle of the thigh was measured using MRS as a secondary endpoint in Study 48. At 18 months, patients receiving givinostat demonstrated a
smaller mean increase in the VL muscle fat fraction (VL MFF) compared to patients who
received placebo. This pharmacodynamic effect of givinostat showing reduced loss of lean
muscle mass is consistent with the purported mechanism of action of the drug and provides
biologic plausibility that the drug is having its intended effect on the muscle, which helps to
confirm the Study 48 findings of a treatment benefit in patients with DMD.
Reference ID: 5351242
NDA

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