Welcome! You are invited to join a webinar: FDA Listening Session: COVID19 Impact on Rare Disease Communities. After registering, you will receive a confirmation email about joining the webinar.

How is the pandemic affecting people living with rare diseases and their families? How does the U.S. Food and Drug Administration (FDA) respond to ensure that the needs of the rare disease community are met with respect to clinical trials, drug shortages and access to personal protective equipment (PPE)? National Organization for Rare Disorders (NORD) and FDA will co-host a virtual “listening…

New Video Series on the 5 Steps of the Drug Development Process and How RDCA-DAP Can Help – NORD (National Organization for Rare Disorders)

There are five steps in the drug development process, which are designed to help ensure that potential new therapies are both safe and effective. Developing medical products for rare diseases encounters many challenges throughout this process. The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is being developed by NORD and the Critical Path Institute (C-Path) to Read more >

Blood Podcast

Blood Podcast Blood Podcast   The editors of Blood are pleased to introduce a new podcast series, published weekly in conjunction with each issue of Blood. Each episode will highlight selected content from recent articles published in Blood.   Subscribe today to receive notification of each new episode as it’s published. Subscribe…

Accelerating Time to Diagnosis: The Global Commission to End the Diagnostic Odyssey for Children With a Rare Disease | CheckRare

The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex challenges impacting the rare disease community.

GRIDS 2020: Managing SARS-CoV-2 Infections in Patients with Lysosomal Disorders

Uma Ramaswami, MD, Royal Free NHS, London, UK talks about the outpatient management, inpatient treatment, and long term follow-up of SARS-C0V-2 infections in patients with lysosomal storage disorders.

This presentation was part the live GRIDS 2020-Virtual Summit held July 1, 2020 and is now available exclusively on CheckRare. To view the entire session and receive CME credit, go to https://checkrare.com/learning/p-grids2020-module4-lysosomal-disease-management-and-covid-19/