NORD Breakthrough Summit

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.

As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.