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…
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 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…
SRNA is joined by Dr. Cynthia Wang and Dr. Lana Harder for an Ask the Expert podcast on “Behavioral and Psychological Changes in Demyelinating Conditions.”
KIF1A.ORG is dedicated to improving the lives of those affected by KIF1A Associated Neurological Disorder and accelerating research to find a cure.
The U.S Food and Drug Administration (FDA) is hosting a public meeting to provide an opportunity for grantees funded as part of the FDA Standard Core Clinical Outcome Assessments (COAs) and Endpoints Pilot Grant Program to share their development plans for the standard core COA sets and to receive feedback from stakeholders.
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.
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/
Three months ago, the epidemiologist weighed in on what we must do to defeat this new threat. We went back to ask: How are we doing, and what comes next?