About ACCESS DMD™

ACCESS DMD™ is an expanded access program for children, adolescents, and adults in the U.S. diagnosed with DMD.

  • The ACCESS DMD™ program will enable participating physicians to obtain and provide deflazacort*, an investigational medication for eligible U.S. patients diagnosed with DMD, while it is under development.
  • Expanded access is a general term often used to refer to a variety of programs available to provide investigational treatment access to patients with life-threatening or severely debilitating disease for whom commercially available therapies have failed or are not available. The distinction between an expanded access program and a clinical trial is that the use of an investigational medication in an expanded access program is intended for treatment, whereas the use of an investigational medication in a clinical trial is intended to answer questions about the safety and effectiveness of the investigational medication.
  • The FDA has authorized the use of deflazacort* for eligible patients with DMD under the ACCESS DMD™ expanded access program. Patients participating in ACCESS DMD™ may only receive deflazacort* through a physician participating in the program. To check to see if your physician who treats your DMD is participating in ACCESS DMD™, click on the list of participating physicians. To request your physician who treats your DMD become a participant in ACCESS DMD™, you may access the patient-to-physician letter here.
  • If you are interested in learning more about expanded access programs, take a look at Expanded Access Program Information for Patients.
  • While participating in ACCESS DMD™, deflazacort* will be provided to participants at no cost.
  • Once ordered by an ACCESS DMD™ participating physician, deflazacort* will be sent directly to enrolled patients or their caregivers. Costs for other tests or routine medical care will need to be covered by the patient’s insurance or at the patient’s expense and are not reimbursed by the ACCESS DMD™
  • Not every patient with DMD who wishes to take part in ACCESS DMD™ will meet the entry criteria.
  • As with any investigational medication that has not been approved by the FDA, deflazacort* may or may not be effective in treating your DMD and there may be risks associated with its use. You will need to talk to your physician who treats your DMD about the possibility and risks of taking part in the program.

*Deflazacort is an investigational medication that has not been approved by the Food and Drug Administration (FDA) and is therefore not proven to be safe and effective.

Eligible patients must:

  • Have a formal diagnosis of DMD
  • Be at least 5 years of age
  • Weigh at least 13 kg
  • Be up to date with all childhood vaccinations, including chicken pox (varicella)

What about side effects?

There is a possibility that some participating patients will experience side effects from deflazacort*. Your physician will discuss the potential side effects with you before obtaining your consent to participate in this program. You should talk to your physician about all of the known side effects of deflazacort*, the possibility of unexpected side effects associated with deflazacort*, and ask any further questions you may have before you agree to participate in ACCESS DMD™. Patients and their caregivers are strongly advised to talk to their physician if there are any concerns about any health changes or side effects while participating in ACCESS DMD™.

What should I do next?

If you are interested in ACCESS DMD™, follow these simple steps:

1) Check the criteria – eligible patients must:

  • Have a formal diagnosis of DMD
  • Be at least 5 years of age
  • Weigh at least 13 kg
  • Be up to date with all childhood vaccinations, including chicken pox (varicella)

2) Check the list of participating physicians to see if your physician is listed

3) If your physician is listed as being participating in ACCESS DMD™, please speak to him or her directly about participating in the program.

Or

If your physician is not listed as participating in ACCESS DMD™, you can print the Patient to Physician Letter in the Resources section and bring it to your physician’s office or download it and email the letter to your physician to help express your interest in the program. Your physician must go through several steps before becoming a participating physician in ACCESS DMD™. It may take a few weeks or longer for your physician to become a participating physician in ACCESS DMD™.

4) If your physician determines you are eligible you will be asked to sign an Informed Consent form. The consent form will provide details about the ACCESS DMD™ program, your rights as a participant, potential side effects and other information about deflazacort*. After you sign the consent form, your physician will advise you once you have been enrolled and will inform you regarding next steps and the anticipated timing for receipt of deflazacort* through ACCESS DMD™. If you are already receiving deflazacort* by other means (not through ACCESS DMD™), you should consult with your physician first before stopping shipments in order to ensure there is no interruption in your treatment.

*Deflazacort is an investigational medication that has not been approved by the Food and Drug Administration (FDA) and is therefore not proven to be safe and effective.

Ready to get started?

LIST OF PARTICIPATING PHYSICIANS