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About The Study

Purpose of Study:

To provide therapeutic treatment for children suffering from Duchenne Muscular Dystrophy (“DMD”). To obtain usable data and subjective/objective feedback from patients and guardians/parents which will in turn be provided at pre-determined intervals to the DMD community.


Study/Treatment Protocol:

  • Infusions and injections of P.C.F.R (Project Care for Rare) compound, approximately commensurate with patient weight per kg.

  • Ongoing assessment of physical abilities utilizing J-tech testing.

  • Muscle biopsy of selected children pre and post treatment to utilize staining testing in identifying dystrophin. 

  • Weekly contact between parents of children in the study and Study coordinators, Jamie from Fight for Alfie or Steve from Wishes for Drew, to keep updated with any changes/improvements.

  • As much as feasible, keeping of a journal or a record of the child in the study throughout the 12 months leading up to publication of result;, tracking changes, improvements, declines, etc. Also tracking of parent/guardian observations and feelings regarding the treatment.




The costs outline below is per child per treatment.

As part of the study, each child will be required to receive treatment every 3 months for a total of 1 year​.


A Single P.C.F.R Compound Infusion

Approx. $4,900

(This varies per child, as treatment is weight dependent)

Doctor Costs, Office Time, & Other Infusion Costs


Physio & Testing Costs



Approx. $100 - $1,500

(Depends on home location of each child)


Approx. $100 - $2,000

(Depends on home location of each child)


Approx. $8,800 - $12,100