Innovations in Care

We provide programs and services that transform the lives of people affected by neuromuscular disease.

Outside Organization Programs & Information

As part of our support to the neuromuscular disease community, MDA posts information provided by outside companies and organizations that are educational or research in nature.

The information, programs and resources listed below are not developed,  sponsored, funded or endorsed by MDA.  The companies and organizations posting below are responsible for all content and information provided. Any questions or queries, please email us at ResourceCenter@mdausa.org. Posting may not be for fundraising or commercial purposes.

Take on Thymidine Kinase 2 deficiency (TK2d)

Whether you're living with an MDS, have been diagnosed with TK2d or are a parent, caregiver, or healthcare professional (HCP) who wants to learn more about MDS and TK2d, visit www.tk2d.com for more information. We can all take on TK2d together.

Gene Therapy Patient Education

The American Society of Gene and Cell Therapy has a variety of educational resources available on their website. Visit https://www.asgct.org/education to learn more about the basics of gene therapy, the clinical trial process and the potential of gene therapy for specific diseases including SMA and MTM.

Pompe Disease Clinical Trial Perception Survey

A biotech company that conducts research and develops treatments for people living with rare diseases such as Pompe disease wants your help. They are recruiting Pompe patients and caregivers to take a short online survey about your knowledge and understanding of clinical trials and research.

The results of this survey will help guide the development of patient support and awareness programs for current and future clinical trials. In appreciation of your time, the summary of the results of this survey will be shared with surveyors and patient advocacy organizations via email. The link to the survey is below:

https://pompediseasesurvey.com

We appreciate you sharing your opinion and experiences to help shape the future of Pompe clinical trials. Better treatment options are common goals across our community, and your voice is essential to progress toward new therapies.

If you have questions about the survey or technical concerns, please email Emily Cortez at PEPTrials at emily.cortez@the-pep.com.