March 26, 2020March 26, 2020
Design Therapeutics Launches with $45M to Advance Therapy Candidates for FA and Other Disorders

Design Therapeutics has launched with $45 million in funding to advance its lead candidate for the treatment of Friedreich’s ataxia into clinical development, ... Read more
March 25, 2020March 23, 2020
Thank You for Your COVID-19 Response — from a Rare Disease Patient

It’s finally happened: the first outbreak within my parish (or “county” for you non-Louisiana folk) has been reported at the time of writing this ... Read more
March 24, 2020March 23, 2020
Don’t Forget About Self-care During the Pandemic

There’s never a “good time” for the world to face a pandemic, or for a region or community to deal with such a significant ... Read more
March 23, 2020March 23, 2020
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more
March 23, 2020March 20, 2020
What We Can Take Away from the Coronavirus Pandemic (So Far)

In times like these, I wish I had superpowers or a magic wand to make everything better. The fear and panic caused by the ... Read more

News

Neurofilament Proteins Show Promise as Biomarkers of Neurodegeneration in FA
March 19, 2020March 19, 2020
Neurofilament light chain (NfL) and phosphorylated neurofilament heavy chain (pNfH) — two key components ...
Columns, My Darling Disability - a Column by Kendall Harvey

I Have a Complex Relationship with Physical Therapy
March 19, 2020March 18, 2020
I was an athlete until my mid-20s. I swam competitively for 14 years and ...
Columns, Little Victories - a Column by Matthew Lafleur

My Physical Therapy Progress: Mastering the Dead Man’s Crawl
March 18, 2020March 18, 2020
At my latest physical therapy appointment, I was out of my wheelchair and on ...
Columns, Fighting FA - a Column by Frankie Perazzola

My Diagnosis Changed Many of My Friendships
March 16, 2020March 12, 2020
Thanks to Friedreich’s ataxia (FA), I have experienced the stress and turmoil of trying ...
News

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
March 12, 2020March 12, 2020
The number of treatments for children with rare diseases has grown over the past decade, ...
News

Worsening Physical Impairments Affect Quality of Life in FA, Study Shows
March 12, 2020March 12, 2020
Physical impairments that worsen with disease progression are the main contributors to a poor ...

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I Am Redefining Hope, Every Day
March 11, 2020March 9, 2020
I was giddy that day, a month or two ago. So much so that ...
Not All Decisions Should Be Rooted in Money
March 10, 2020March 9, 2020
I feel like I’ve recently made a huge decision. To some, my choice may ...
Podcast Series Promotes FA Advocacy
March 6, 2020March 5, 2020
Exenatide Therapy Increases Key Frataxin Protein Levels, Study Finds
March 5, 2020March 5, 2020
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
March 4, 2020March 5, 2020
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
March 3, 2020March 3, 2020

Easier Doesn’t Always Mean Better
March 3, 2020February 28, 2020
The podcast I co-host, “Two Disabled Dudes,” is celebrating a major milestone this week: ...
Hope for a Friedreich’s Ataxia Cure Keeps Fear of the Future at Bay
March 2, 2020February 27, 2020
Dealing with Mental Health Issues While Living with a Chronic Illness
March 2, 2020February 27, 2020
Accessible Stalls Are More Than a Convenience
February 28, 2020February 26, 2020

HIV Therapy Etravirine Set to Enter Phase 2 FA Trial in Italy
February 27, 2020February 28, 2020
Etravirine — an approved anti-viral therapy for HIV — is expected to soon enter ...
Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
February 27, 2020February 27, 2020
New US Institute for Gene Therapies Seeks to Modernize Regulatory, Reimbursement Framework
February 27, 2020February 26, 2020

Columns, Little Victories - a Column by Matthew Lafleur

I Might Be Rare, but I Am Not Alone
February 26, 2020February 24, 2020
My teacher and my best friend’s mom talked amid the gaggle of parents picking ...
Columns, No Good Excuse - a Column by Sean Baumstark

Learning a Lesson in Customer Service from a Recent Airline Experience
February 25, 2020February 21, 2020
If you follow me on social media, you probably saw this column coming. In ...
Friedreich's Ataxia, News

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
February 21, 2020March 3, 2020
An abundance of events are afoot around the world to mark Rare Disease Day ...
A Practical Guide to Life with FA - a Column by Christina Cordaro, Columns

FA Community Bonds Are Strong Even When We’re Far Apart
February 21, 2020February 19, 2020
I remember meeting my dear friend Mary for the first time. Mary also has ...
News

Increased Thickness of Heart’s Left Ventricle, Linked to Genetic Disease Severity in Adults with FA
February 20, 2020February 20, 2020
Increased thickness of the lower left chamber of the heart — the left ventricle ...
Columns, Little Victories - a Column by Matthew Lafleur

The Zeego Tales: A Conflicting Gift
February 19, 2020February 17, 2020
I saw one of my middle school teachers the other day. Two decades have ...
Columns, No Good Excuse - a Column by Sean Baumstark

Recovery Has Reframed My Priorities
February 18, 2020February 14, 2020
I’m realizing that short-term disability insurance provides value beyond the financial benefit. Of course, ...
Columns, Fighting FA - a Column by Frankie Perazzola

Periods of Isolation Allow Me to Thrive
February 17, 2020February 13, 2020
I have been in many social situations where I have blown it. Almost every ...
A Practical Guide to Life with FA - a Column by Christina Cordaro, Columns

Rare Disease Day 2020: How You Can Get Involved
February 14, 2020February 13, 2020
Did you know that one in 20 people will live with a rare disease ...

October 5, 2017February 20, 2019

Urinary, Bowel, Sexual Symptoms in FA Lead to Lower Quality of Life, Study Reports

News

Many Friedreich’s ataxia patients experience urinary tract symptoms, bowel problems, and sexual dysfunction, which leads to a lower quality of life, research shows. The study, “Urinary, bowel and sexual symptoms in a cohort of patients with Friedreich’s ataxia,” was ... Read more

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