Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.
Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.
Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare Disorders
As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
Improving Access to Rare Disease Care
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
Bridging the Gap: Education, Awareness Key to Translating Medical Innovation to Community Health Care in Rare Diseases
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
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