For decades, NORD has pursued federal and state policies that will change the lives of Americans impacted by rare diseases for the better. NORD’s work can be viewed below. This page is updated daily to provide timely information. Please contact us if you have questions or need additional information.
NORD Submits Comments to the National Center for Advancing Translational Sciences (NCATS) RFI on Facilitating the Early Diagnosis and Equitable Delivery of Gene-Targeted Therapies to Individuals with Rare Diseases
Published: Jun 30, 2021
Tagged in: Gene Therapies, Advancing Rare Disease Research and Regulatory Science
FDA’S Accelerated Approval Pathway: A Rare Disease Perspective
Published: Jun 30, 2021
Tagged in: Access to Innovative Medicines and Therapies
NORD Submits Comments on FDA Draft Guidance: Submissions for Individualized Antisense Oligonucleotide Drug Products
Published: Mar 8, 2021
Tagged in: Access to Innovative Medicines and Therapies, Advancing Rare Disease Research and Regulatory Science
NORD submits comments to FDA on Request for Information on Rare Disease Clinical Trial Networks
Published: Jul 31, 2020
Tagged in: Advancing Rare Disease Research and Regulatory Science, Rare Disease Clinical Trials, Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)
NORD submits comments on FDA Draft Guidance on Interpreting Sameness for Gene Therapy Products Under the Orphan Drug Regulations
Published: Jul 28, 2020
Tagged in: Gene Therapies, Advancing Rare Disease Research and Regulatory Science, Orphan Drug Act
