The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed. 

Published in Human Gene Therapy, 25 Nov 2020

Because many of the major targets of long-term therapy for FA are in the central nervous system (CNS), FARA worked with several FA investigators to write an in-depth review of the pathology present in the brain and spinal cords of patients with FA. The diverse group of experts was able to evaluate evidence gathered from human clinical observations, physiological, pathological and imaging approaches, as well as studies in animal models. The resulting paper provides detailed summary of the current understanding of what areas of the spinal cord and brain are impacted by the disease, including what specific cells are vulnerable to the loss of frataxin.

Moreover, the review also provides some insight into the temporal course of neuropathological changes in the CNS, which helps to provide information on areas that may respond to therapy and be successfully treated at different timepoints during the progression of the disease.

This comprehensive review of the impact of FA in the CNS is useful to patients, caregivers, physicians, as well as drug developers, especially those planning to test therapeutics aimed at the spinal cord and/or brain. FARA effort to organize and support production of this review represents the ongoing commitment to focusing resources on filling gaps in our understanding of FA and building relationships needed to find effective treatments.

To access the full publication click HERE.

The FARA Energy Ball took on a virtual form this year featuring a live broadcast and an online auction. An estimated 550 viewers tuned in to the broadcast that featured talks from FARA and local Energy Ball leadership, highlight videos from FA community members, a Fund a Cure donation call, and a live auction. Emcee Wendy Ryan and Auctioneer Scott Robertson brought their signature energy and sincere support to the event and the cause.

In total, the virtual FARA Energy Ball raised over $1 million to help advance FA research! Thanks to all who tuned in and supported this event.

The Senate passed the resolution designating September 25th, 2020 as National Ataxia Awareness Day! Even though the awareness day has come and gone this year, having the Resolution approved means that it will be much easier for Senators Warren and Hyde-Smith to reissue in the years to come. Thank you to all who met, wrote and called their Senators to make this recognition possible.

Description: Are the holidays on your mind? Are you concerned about continued isolation as the winter months approach? Every year, rare disease community members balance the busy holiday season while also managing an illness. During the pandemic, there may be added challenges caused by the absence of normal traditions and the need to stay COVID-19 safe. Join us for a free, 45-minute webinar to learn how to identify stressors and use coping strategies to stay positive and live your best rare life this holiday season and beyond! 

This webinar is intended for patients, caregivers, advocates, students and the general public.

Registration for Rare Across America is open! This is your opportunity to make an impact on federal policy from home. The two week program will include social media campaigns, an update from the NIH, advocacy training and the opportunity to meet with your members of Congress…all virtual, all happening right in your own house! Don’t miss this amazing opportunity!

Mark you calendars! The EveryLife Foundation for Rare Diseases has announced a big change in programming for 2021. Rare Across America will now take place virtually from Feb 22 - Mar 5 and Rare Disease Week is moving to July 19-22 in Washington, DC.