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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.

Study Costs


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

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