April 19, 2018April 19, 2018

Retrotope Submits Design of Pivotal Trial for Its Friedreich’s Ataxia Therapy to FDA

News

Retrotope has submitted the design of a pivotal clinical trial for its Friedreich’s ataxia therapy RT001 to the U.S. Food and Drug Administration. The submission was prompted by results of a Phase 1/2 trial showing that ... Read more

April 17, 2018April 17, 2018

#AAN2018 – FA Patients Show Gradual Decline in Visual Acuity Over Time, Study Finds

News

Researchers found that patients with Friedreich’s ataxia (FA) show a gradual reduction in low-contrast visual acuity over time. This decline is more pronounced in those with higher numbers of ... Read more

April 16, 2018April 13, 2018

Moving Out Despite Friedreich’s Ataxia

Columns, Fighting FA - a Column by Frankie Perazzola

Following my Friedreich’s ataxia diagnosis in June 2015, one of the first things the doctors told me was that I would progressively lose my ability to live independently and ... Read more

April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

News

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 12, 2018April 11, 2018

Fight or Flight in FA Patients

Columns, My Darling Disability - a Column by Kendall Harvey

Almost everyone can point to moments in their lives dictated by the choice: fight or flight. By definition, the fight-or-flight response is a physiological reaction that occurs in response to a ... Read more

April 10, 2018April 10, 2018

Investigational Friedreich’s Ataxia Therapy RT001 Found to Be Safe, Well-Tolerated in Early Trial

News

Results from a Phase 1/2 clinical study support the safety and tolerability of RT001, an investigational therapy for Friedreich’s ataxia. Patients treated with RT001 also were seen to improve ... Read more

April 9, 2018April 6, 2018

My Experience with Advocacy in Baltimore

Columns, Fighting FA - a Column by Frankie Perazzola

Part of the advocacy realm that I truly enjoy is the opportunity to travel to new places and speak to different groups of people. At the beginning of March, ... Read more

April 5, 2018April 5, 2018

Mobility Devices Linked to Worse Quality of Life in Pediatric FA Patients, Study Suggests

News

Researchers found that the use of mobility devices is associated with a poorer quality of life in children with Friedreich’s ataxia (FA). The study, “Impact of Mobility Device Use ... Read more

March 29, 2018March 29, 2018

P38 Protein A Potential Therapeutic Target for Friedreich’s Ataxia, Study Shows

News

Inhibitors of p38 — a protein involved in regulating cell growth — show potential for use as therapeutic agents in the treatment of Friedreich’s ataxia (FA), a recent study ... Read more

March 28, 2018March 27, 2018

Please Don’t Refer to My Caregivers As ‘Slaves’

Columns, Through My Eyes - a Column by Jamie-Lee Dwyer

When I was a little girl, I used to have this recurring fantasy every so often in which I was Queen of I don’t even know what, with all ... Read more

March 27, 2018March 28, 2018

Study Describes Pitfalls of FA Genetic Tests, Possible Need for More Analysis

News

Researchers report that genetic tests for Friedreich’s ataxia (FA) are complex and may require additional screening in cases of typical clinical presentation. The study, “Pitfalls in molecular diagnosis of Friedreich ... Read more

March 22, 2018March 20, 2018

What rideATAXIA Means to Me

Columns, My Darling Disability - a Column by Kendall Harvey

Friedreich’s ataxia (FA) is a big, overwhelming disease. And with it being so rare, it can feel very isolating. My immediate family and I had never even heard of ... Read more