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News & Press Releases

Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.


Award for Innovative Mindset

FARA, the CureFA Foundation, and fara Australia are proud to announce the recipients of the Award for Innovative Mindset (AIM). AIM was designed to promote the exploration of high-risk, high-gain, and potentially groundbreaking concepts in FA research. The request for proposals was initiated in May, during Friedreich's Ataxia Awareness Month, and the research awards are supported in part by funds raised in the Lend Us Some Muscle campaign. The intent of the award was to inspire creativity, thoughtful reflection, innovation and to advance FA research, during a time when many investigators were away from their labs and daily routines due to the shutdown. Seventeen applications were received and four were awarded funding. A description of each project follows below.


Sanjay Bidichandani
Sanjay Bidichandani, PhD - University of Oklahoma
Single Cell Gene Expression Analysis in Friedreich Ataxia This application proposes a new model to explain the (apparently) high residual level of frataxin seen in FA patients. The current understanding is that FA develops when cells express levels of frataxin protein that are 10‐20% of normal levels. Thus, when compared to other loss-of-function conditions, where disease is typically triggered at residual protein levels/activity of <5%, FA seems to manifest at relatively higher residual protein levels. The hypothesis put forward in this grant proposal is that most FA cells actually express very low levels of frataxin (<5%, as in other recessive disorders), and that a substantial minority of cells show “escape from gene silencing” and thus express near normal levels of transcript due to FXN genes that, despite containing an expanded GAA repeat, are not fully silenced. This group plans to test this hypothesis by analyzing FXN expression and silencing in individual cells. This model has important implications for the understanding of FA pathogenesis, and for the development of treatments.
Cosponsor: CureFA Foundation and fara Australia

Christina Cortez-Jugo
Christina Cortez-Jugo, PhD - University of Melbourne, Australia
Targeted Delivery of Frataxin DNA to Proprioceptors of the Dorsal Root Ganglia
This application proposes to use nanoparticles instead of viruses to deliver the frataxin gene in gene therapy approaches. Nanoparticles are small aggregates made of polymers, lipids and other molecules that can deliver a specific cargo (in this case the frataxin gene) to cells. This investigator proposes to improve the delivery of the cargo to the specific cell types that are mostly affected in FA, by coating the nanoparticle surface with antibodies and ligands that will promote their binding and uptake by the target cells. The ability to target specific cells affected in FA could increase the concentration of the therapeutic where it is needed, and reduce dosage, cost of treatment and side effects.
Cosponsor: CureFA Foundation and fara Australia

Giovanni ManfrediHelene Puccio


Giovanni Manfredi, MD, PhD - Weill Cornell Medicine & Helene Puccio, PhD - Institute NeuroMyoGène, Lyon, France
Mitochondrial integrated stress response in FA cardiomyopathy
The hypothesis of this proposal stems from the observation that death from cardiomyopathy in FA occurs in the third or fourth decade of life, but surprisingly the FA heart often maintains adequate function until shortly before death. This suggests that the FA heart is able to adapt, at least initially, to the defects caused by the loss of frataxin. This adaptation likely involves metabolic rewiring to allow the utilization of alternative energy sources and other events. The investigators propose that these events recapitulate the “mitochondrial integrated stress response” (well characterized in other mitochondrial diseases) which is an evolutionarily conserved response designed to help the organisms face periods of acute stress, but if chronic and unresolved, becomes “maladaptive”. This proposal aims to determine whether there is evidence of mitochondrial integrated stress response in the heart of an FA mouse model.
Cosponsor: CureFA Foundation and fara Australia

Seth Masters
Seth Masters, PhD - Walter and Eliza Hall Institute, Melbourne Australia
Targeting neuroinflammation in Friedreich's Ataxia
This grant proposal addresses the role of inflammation in FA. There is evidence of inflammation due to low levels of frataxin in cell lines, in mouse models, and in patients with FA, however mechanistic insights into the trigger for inflammation in FA are missing. This investigator hypothesizes that a specific innate immune pathway drives inflammation in FA. The cellular pathway normally detects virus/bacteria, but is also known to respond to damaged mitochondria. In FA, the damage to mitochondria due to frataxin loss would cause activation of this specific innate immune pathway. The aim of this proposal is to demonstrate mitochondrial damage leading to activation of this innate immune pathway with associated inflammatory biomarkers which may help stratify and direct therapy for FA.
Cosponsor: CureFA Foundation and fara Australia

 

Monthly Update - August 2020


Dear friends,

As you know, Fall is the season for many FARA fundraising events, a scientific conference, and our largest educational FA Patient Symposium. We've been working on adapting those events to a virtual format, and we are excited to deliver all of their energy and information straight to your home. You will find the details for these events throughout this newsletter.

We also recently launched a new program called The Pathway - a monthly recurring gift program. In honor of the 1 in 50,000 people living with FA, we set a goal to enroll 50 new contributors in The Pathway over the next couple of weeks. A monthly donation of $10 or more is a budget friendly way to support ongoing research.

In the first week, 20 new participants enrolled in the program, and I am grateful for the opportunity to be one of them. I believe in the power of research to transform lives. FARA funds research along the entire continuum- from the lab bench to the clinical bedside. Sustaining contributions received through The Pathway allow FARA to ensure that researchers are exploring all molecular, systemic and clinical pathways that will lead to treatments and a cure with urgency. ...

Read the Full Monthly Update
 

FARA Job Posting - Communications Coordinator

Click to view PDF
FARA is seeking a full time Communications Coordinator.

The successful candidate will have professional communications experience, strong writing skills, excellent interpersonal/ teamwork, and a demonstrated interest in the health and human service industry. The candidate will be given the opportunity to show initiative, acquire new skills and grow professionally.

Please click HERE to view the PDF.
 

Third Party Data Breach

Recently, FARA learned of a data breach at Blackbaud Inc., a third-party data service provider we use for our fundraising efforts. FARA was one of many nonprofits and schools that was impacted by this incident.

Blackbaud informed us that they discovered and stopped a ransomware attack and, with the help of independent forensics experts and law enforcement, successfully prevented the cybercriminal from locking them out of the system and taking full control of their files. However, the attackers were able to remove a backup file that contained some personally identifying information about Blackbaud's nonprofit clients' donors and prospective donors, including those of FARA. Please note that no credit card or bank account information was compromised in this breach. Click HERE to read a full description of the incident on Blackbaud’s website.

Blackbaud believes they have successfully retrieved the stolen data. Furthermore, Blackbaud has firmly expressed to us that based on the nature of the incident, the research performed by them, and information provided by third-party investigators and law enforcement, that they do not believe that any data was or will be misused; or will be disseminated or otherwise made available publicly.

Ensuring the safety of our donors’ data is of the utmost importance to FARA and we make every effort to safeguard your information. FARA’s contractual agreements have always required Blackbaud to keep our constituent information confidential and to have security procedures in place to minimize breaches. As part of their ongoing efforts to help prevent something like this from happening in the future, Blackbaud has implemented changes to further protect your data. Such measures include identifying and rectifying the vulnerability associated with this incident, and confirmation testing by multiple third parties, including the appropriate platform vendors, that the fix withstands all known attack tactics.

Although Blackbaud asserts that there is a very low risk from this breach, should you encounter any suspicious activity that you believe may be related, we recommend you promptly report it to us and to the proper law enforcement authorities. If you have any related questions or concerns, please contact us at (484) 879 6160 or info@curefa.org.
 
 

Monthly Update - July 2020


Dear friends,

I hope you and your family are finding ways to enjoy the summer while also staying safe. At FARA, we continue to advance a number of research projects, advocacy initiatives, and fundraising campaigns. I hope the information in this newsletter serves as one indicator of volume of activity still happening even in a new socially distanced reality. Opportunities to participate include clinical trials, the United Against Ataxia Hill Day, the virtual rideATAXIA Global Challenge, the FA Symposium series, the virtual FARA Energy Ball, and contacting your representatives to encourage funding for the National Institutes of Health. Keep engaging your community and keep us informed by posting on social media and tagging FARA using #cureFA.

Thank you for your continued support of FA research. ...

Read the Full Monthly Update
 

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