February 14, 2019February 13, 2019

The Hopes and Risks of Starting a Friedreich’s Ataxia Clinical Trial

Columns, My Darling Disability - a Column by Kendall Harvey

When diagnosed with Friedreich’s ataxia, the first things people usually ask are: “What can I do about this? Is there a treatment or cure? Where do I sign up?” ... Read more

February 14, 2019February 14, 2019

Iron-induced Cell Death May Play Key Role in FA, Study Suggests

News

Iron-induced cell death may contribute to the progression of Friedreich’s ataxia, a study in human and mice cells shows. Inhibition of this degenerative mechanism may represent a new therapeutic ... Read more

February 14, 2019February 14, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 13, 2019February 12, 2019

The Anchor on My Wheelchair from My Steadfast FA Friend

Columns, Little Victories - a Column by Matthew Lafleur

A small wooden trinket hangs on the back of my wheelchair. It’s a navy blue anchor with hand-painted white letters: “Cure” is written down the anchor’s stem and “FA” ... Read more

February 11, 2019February 14, 2019

When in Doubt About Social Media Content, Just Be Honest

Columns, Fighting FA - a Column by Frankie Perazzola

Almost every day, I have a conversation with myself about the level of my own vulnerability on the internet. Is this comment too much? Should I discuss this topic? ... Read more

February 8, 2019February 14, 2019

The Power Behind the FARA Patient Registry

A Practical Guide to Life with FA - a Column by Christina Cordaro, Columns

Last month, I attended a PTC Therapeutics meeting with two other Friedreich’s ataxia patients named Kyle Bryant and Connor Sweeny, plus people who work for the Friedreich’s Ataxia Research ... Read more

February 8, 2019February 14, 2019

Neurocrine, Voyager Collaborating on VY-FXN01 Gene Therapy for Friedreich’s Ataxia

News

Neurocrine Biosciences and Voyager Therapeutics have formed an alliance in hopes of bringing potentially life-changing treatments to market for Friedreich’s ataxia (FA) and Parkinson’s disease. The collaboration is aimed ... Read more

February 7, 2019February 14, 2019

When Life Hands You Lemons

Columns, My Darling Disability - a Column by Kendall Harvey

“Life isn’t fair.” “No one said this would be easy.” We have all heard these platitudes and we’ve all repeated them. And yes, there is truth in these well-worn ... Read more

February 6, 2019February 13, 2019

In the Waiting Room with FA

Columns, Little Victories - a Column by Matthew Lafleur

Before I had a smartphone to help me pass the time, I was OK. I was easily entertained in waiting rooms, whether I read outdated magazine articles or focused ... Read more

February 1, 2019February 12, 2019

The Cure to Being Burdened by Shopping for Food

A Practical Guide to Life with FA - a Column by Christina Cordaro, Columns

I used to enjoy going to the grocery store. I loved picking out items, seeing what’s new in stock, and hunting good deals in the store’s weekly ads. However, ... Read more

January 31, 2019February 12, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

News

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal ... Read more

January 31, 2019February 12, 2019

Omaveloxolone Safe, Improves Neurological Function in FA Patients, Phase 2 Trial Shows

News

A 12-week treatment with the investigational therapy omaveloxolone improved neurological function, and was safe and well-tolerated by Friedreich’s ataxia (FA) patients in a Phase 2 clinical trial. The study, ... Read more