Investigator-Initiated Studies

A concept is an optional 1–2-page high level summary used for Sarepta’s review and initial recommendation. If Sarepta is interested in the study concept, investigators must submit an application and full study proposal to be considered for funding. Proposals must include study background, objectives, outcome measures, methodology/study design, study duration, budget, milestones, and timelines.

No, submitting a concept form is an optional step which allows Sarepta to make an initial recommendation. If the review committee decides the study is in scope, investigators must submit an application and full proposal through Sarepta’s Web Portal to be considered for funding. Final assessment is based on the application and full proposal.

Proposals must include study background, objectives, outcome measures, methodology/study design, study duration, budget, milestones, and timelines.

Yes, a detailed budget, CV and the institution’s official tax documentation are needed.

To submit a concept form please connect with a Sarepta Medical Affairs team member or email IIS@sarepta.com. To submit a full proposal, please click here.

Clinical studies (including observational studies) and non-clinical studies in rare neuromuscular diseases are eligible. Please note, proposals to develop databases or registries without defined research objectives or those that are not designed to address a valid scientific question are typically not supported by the IIS program. If you have any questions regarding study eligibility, please email IIS@sarepta.com or connect with a Sarepta Medical Affairs team member. 

For requests related to compassionate use, please refer to our Compassionate Use Policy for more information.

Applications are reviewed monthly, and feedback is provided within 90 days.

Sarepta’s subject matter experts and IIS committee critically review proposals and evaluate them based on scientific merit and alignment with Sarepta’s strategic areas of interest.

The amount of funding provided to each IIS is dependent on the study type. Each study is evaluated independently, and all funding requests are subject to a fair market value assessment. Purchase of capital investments (e.g., equipment, laptops, software) are not supported.

Approved proposals are based on milestone payments. If you have any additional questions regarding payment, please contact IIS@sarepta.com.

Investigators will be responsible for the following (as applicable),  including but not limited to:

• Developing a research protocol and obtaining IRB approval
• Maintaining rigorous standards for research integrity and data quality
• Providing regular study updates to the Sarepta team and a written report upon study completion
• Committing to disclosing and disseminating the findings in an appropriate, transparent, and timely manner in accordance with agreed timelines
• Ensuring compliance with all necessary safety reporting requirements by Sarepta and local/national health authorities