The Muscular Dystrophy Association supports the Patient Access Act, introduced last week by U.S. Representatives Brett Guthrie (R-KY) and Nanette Barragán (D-NY).
 

The Patient Access Act would allow manufacturers of crucial therapies to assist patients in their travels and lodging to receive treatment. This bill could greatly improve access to therapies for the neuromuscular disease (NMD) community, especially as gene therapies continue to be developed. While gene therapies are administered only once and can be transformative, they are also expensive - and require extensive monitoring to ensure the safety of the patients who receive them for approximately the first year after treatment administration. Supplying this support to patients and their families will play a meaningful role in reducing what is an already significant financial burden.
 

This bill will also support the rare disease community by boosting access to the few specialists able to administer gene therapies or other treatments. Patients and their families are often left needing to travel long distances to receive needed care, and this bill will ease that burden.
 

MDA thanks Representatives Guthrie and Barragán for taking steps to increase access to care for the NMD community.