Rare Disease Advocacy

The GCA is pleased to announce that we have secured an FDA Patient Listening Session for neuronopathic Gaucher disease (nGD). The GCA will be bringing type 3 Gaucher families to meet with FDA Regulators in the fall of 2021 on the need for approved treatments for nGD. Let us know if you’d like to find out more.

The GCA is involved with numerous advocacy efforts including Rare Disease Week on Capitol Hill, state level efforts on co-pay accumulators, insurance patient denials and appeals, FDA patient advisors, and many more. The GCA is able to engage in advocacy on behalf of our community due to the generous sponsorships of our industry partners at AVROBIO, Sanofi and Takeda. With our industry partners assistance, the GCA is able to respond to community advocacy needs as they arise.