Sarepta is committed to developing innovative therapies for patients. We take our responsibility to act as a true partner with patient communities very seriously and are committed to ensuring our innovations reach as many patients as possible, as quickly as possible.

Rett Syndrome (University of Massachusetts/StrideBio)

SareptAssist patient support program

SareptAssist is a patient support program designed to provide individuals with the information to navigate the process of starting and staying on therapy. This program is for individuals residing in the United States who are eligible for treatment with a Sarepta product. A dedicated case manager with experience in rare diseases, insurance plans, and healthcare networks will be your partner.

You’ll receive information on:

  • insurance benefits
  • financial assistance options
  • treatment logistics
  • options for weekly infusions
  • ongoing education and support

Questions about SareptAssist?

Just give us a call at 1-888-SAREPTA (888-727-3782)

Case managers are available Monday—Friday, 8:30 am to 6:30 pm ET.

Spanish-speaking case managers and interpreters for other languages are available. SareptAssist is a resource available only to those who have been prescribed a Sarepta therapy. SareptAssist is only available in the US.

Managed Access Program

Sarepta is committed to developing innovative therapies for patients with Duchenne muscular dystrophy (DMD). We take our responsibility to act as a true partner with patient communities seriously, and we are committed to working with physicians who have determined that their patient has an unmet medical need.

We believe that well-designed clinical trials are the best way to determine the safety and effectiveness of investigational therapies and for patients to gain access prior to regulatory approval. However, we understand that patients and families may be interested in accessing our investigational therapies prior to regulatory approval, and outside of the clinical trial setting, through managed access programs (also known as an early/expanded access, or named patient program).

A managed access program (MAP) is a mechanism through which physicians can prescribe, within their professional responsibility, an unapproved treatment for patients. Use of an unapproved treatment is based on physicians’ informed clinical assessment to determine whether there is a special need for the unapproved treatment because (a) there is no approved treatment available or (b) available treatments are not suitable for that patient. For this MAP, physicians will need to assess patient eligibility by determining whether the patient meets pre-specified criteria, including pre-specified medical, regulatory and other criteria, including financial funding. 

Compassionate Use Policy

Sarepta is committed to developing innovative, safe and effective therapies for patients with Duchenne muscular dystrophy (DMD). We take our responsibility to act as a true partner with the Duchenne community very seriously, and are committed to ensuring our innovations reach as many patients as possible, as quickly as possible.

We understand that patients and families are interested in accessing our DMD exon-skipping investigational therapies prior to regulatory approval, and also outside of the clinical trial setting, through compassionate use programs.* Compassionate use and other mechanisms for pre-approval access to our DMD exon-skipping agents must balance our shared urgency with many other factors. These include regulatory and practical considerations, such as each agent’s benefit/risk profile, the impact on clinical development, and the feasibility of providing fair and sustainable access.

After much consideration, we have determined that, at this point in time, we are unable to offer a compassionate use program on a fair and sustainable basis for any of our investigational agents without jeopardizing our ability to provide patients broad, sustainable, and long-term access. We believe that the best path to potential regulatory approval, and subsequent access to the greatest number of patients, is by executing our current and future clinical trials as efficiently as possible.

We will continue to evaluate the possibility of compassionate use and pre-approval access mechanisms. We will update the Duchenne community if any such programs can be conducted in a fair and sustainable way without compromising clinical development and potential regulatory approval of any of our DMD exon-skipping investigational therapies.

Should patients, families, or physicians have further questions or require additional information about the status of our exon skipping therapies, please contact our Global Medical Information department by email at [email protected] or in the United States toll free at 1-888-727-3782, option 2.

*Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation.