Stem Cells, Inc is actively pursuing a Phase II PMD clinical trial. The PMD Foundation’s Scientific Advisory Board and I are in contact with both Stem Cell’s, Inc and the Principal Investigator for the Phase I trial. They recently submitted the final Phase I PMD Safety Trial report to the FDA and are waiting on the FDA review and approval. While they are waiting, Stem Cells, Inc has started the laborious process of planning a Phase II Efficacy protocol and plan. Once the plan is complete, the FDA must approve the plan before it can be implemented. This is NOT to say that a Phase II trial can or will be conducted, there are many obstacles to navigate. The plan they are developing involves: 1) the specific conditions for patient enrollment: such as age, genetic diagnosis, MRI results, clinical diagnosis and severity; 2) The number of patients that must be enrolled before it can proceed; 3) Identification of one or more physical locations and Principal investigators where the trial will be conducted and 4) the ability of the patient and family to withstand the rigors of the trial (such as travel, time away from home, and monitoring). NOTE: The most important work that the PMD Foundation, and all of the PMD community, must do is to create an effective Patient Registry. With this available, it will be much easier and quicker for the PMD community to help the Principal Investigators identify which patients meet the Phase II trial requirements and determine who is willing to be contacted to become fully informed before making the decision to participate in the trial.
Further, the Registry will also serve to support patient enrollment in future Clinical Trials and other PMD research projects. For example, there are currently two PMD studies being conducted at Wayne State University which would benefit greatly from the contact data contained in a Patient Registry. We are also now in contact with a consortium of clinicians and researchers from around the world who WANT to help us (including Harvard, Stanford, Mayo Clinic, Cleveland Clinic, Kennedy Krieger, Baylor, University of Pittsburgh, Children’s National in Washington DC, Oregon, OHSU, U Utah, U Rochester, U Wisconsin-Madison, U Nebraska, U Colorado and internationally in Australia, Canada and Amsterdam). We must do everything we can to facilitate these activities. This is a new project, and even though you may think that the Foundation already has your contact information, we cannot verify that it is up to date and we do not have permission to reach out to you. The participation of our PMD families is vital. If WE can’t find you, then THEY can’t help you! Please, the urgency has never been more critical than now. We don’t want the scientific community to walk away because they can’t find enough PMD patients.
What do we need?We need to take the first step toward creating a PMD Patient Registry by collecting contact information for all those who are interested in participating in future clinical trials and research projects.Please send the following ASAP to: email@example.com
your name, email address and phone number your PMD child(ren)’s name(s) and birthdate(s) – including all living or deceased (with date of death.) Copy and paste this next sentence into the email, if you agree: I have provided my contact information, and hereby grant representatives of The PMD Foundation permission to contact me to gather data for the Patient Registry, notify me about available research participation and update me on other matters related to PMD.
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