January 22, 2021January 21, 2021
NORD’s Caregiver Respite Program Continues Through Pandemic
News, syndicated
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, ... Read more
January 21, 2021January 20, 2021
How I Get Through Bad Days With Friedreich’s Ataxia
Columns, My Darling Disability - a Column by Kendall Harvey
Friedreich’s ataxia (FA) is a cruel, relentless, lifelong battle. It can make good days hard and bad days ... Read more
January 21, 2021January 21, 2021
FXN Gene Therapy Treats Heart Problems in New FA Mouse Model
News
A gene therapy that delivers a healthy version of the FXN gene can correct heart problems in a new ... Read more
January 20, 2021January 18, 2021
Crawling Toward Fitness as My Abilities Change
Columns, Little Victories - a Column by Matthew Lafleur
My arms couldn’t hold my body up in a crawling position any longer, so I face-planted on the matted ... Read more
January 14, 2021January 12, 2021
The FA Community Needs Your Help to Advance a Potential Treatment
Columns, My Darling Disability - a Column by Kendall Harvey
The Friedreich’s ataxia (FA) community has been abuzz with excitement recently. The first two phases of a clinical ... Read more
January 14, 2021January 14, 2021
First In-human Trial of XCUR-FXN Planned for 2022
News
Exicure recently discussed preclinical research data and the next steps in developing XCUR-FXN, its investigational antisense oligonucleotide (ASO) therapy for ... Read more
January 13, 2021January 8, 2021
Remembering Keith Andrus, Who Left His Mark on the FA Community
Columns, Little Victories - a Column by Matthew Lafleur
Sticking to a New Year’s resolution may better our lives in the short-term, but thinking about what I will ... Read more
January 12, 2021January 8, 2021
Finding a Silver Lining in the Wake of 2020 and the Pandemic
Columns, No Good Excuse - a Column by Sean Baumstark
It’s obvious that things don’t change simply because we turn the page on another calendar year. Typically, the early ... Read more
January 12, 2021January 11, 2021
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum
News, syndicated
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to ... Read more
January 7, 2021January 5, 2021
My Loved Ones Perceive My Symptom Progression Differently
Columns, My Darling Disability - a Column by Kendall Harvey
Over the holidays, we spent time with family that we don’t see regularly. I get so wrapped up in ... Read more
January 7, 2021January 7, 2021
FA Research Alliance Petitioning for Urgent Access to Omaveloxolone
News
With backing from a petition that is now circulating online, the Friedrich’s Ataxia Research Alliance (FARA) will ask Reata ... Read more
January 6, 2021January 4, 2021
COVID-19 Vaccines Bring Relief for Many, Envy for Some
Columns, Little Victories - a Column by Matthew Lafleur
Despite whatever successes we saw in 2020, the year mostly will be remembered for a pandemic that affected almost ... Read more

January 22, 2021January 21, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization ... Read more

January 21, 2021January 20, 2021

How I Get Through Bad Days With Friedreich’s Ataxia

Columns, My Darling Disability - a Column by Kendall Harvey

Friedreich’s ataxia (FA) is a cruel, relentless, lifelong battle. It can make good days hard and bad days intolerable. When people tell me I am “inspiring” or ask how ... Read more

January 21, 2021January 21, 2021

FXN Gene Therapy Treats Heart Problems in New FA Mouse Model

News

A gene therapy that delivers a healthy version of the FXN gene can correct heart problems in a new mouse model with cardiac-specific symptoms of Friedreich’s ataxia (FA), a ... Read more

January 20, 2021January 18, 2021

Crawling Toward Fitness as My Abilities Change

Columns, Little Victories - a Column by Matthew Lafleur

My arms couldn’t hold my body up in a crawling position any longer, so I face-planted on the matted floor in defeat, yet with a small, self-satisfied smile. As ... Read more

January 14, 2021January 12, 2021

The FA Community Needs Your Help to Advance a Potential Treatment

Columns, My Darling Disability - a Column by Kendall Harvey

The Friedreich’s ataxia (FA) community has been abuzz with excitement recently. The first two phases of a clinical trial sponsored by Reata Pharmaceuticals showed that the oral therapy omaveloxolone ... Read more

January 14, 2021January 14, 2021

First In-human Trial of XCUR-FXN Planned for 2022

News

Exicure recently discussed preclinical research data and the next steps in developing XCUR-FXN, its investigational antisense oligonucleotide (ASO) therapy for Friedreich’s ataxia (FA), in a live webcast. After initiating investigational ... Read more

January 13, 2021January 8, 2021

Remembering Keith Andrus, Who Left His Mark on the FA Community

Columns, Little Victories - a Column by Matthew Lafleur

Sticking to a New Year’s resolution may better our lives in the short-term, but thinking about what I will be remembered for matters more to me. So, instead of ... Read more

January 12, 2021January 8, 2021

Finding a Silver Lining in the Wake of 2020 and the Pandemic

Columns, No Good Excuse - a Column by Sean Baumstark

It’s obvious that things don’t change simply because we turn the page on another calendar year. Typically, the early weeks of a new year set the stage for considering ... Read more

January 12, 2021January 11, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 7, 2021January 5, 2021

My Loved Ones Perceive My Symptom Progression Differently

Columns, My Darling Disability - a Column by Kendall Harvey

Over the holidays, we spent time with family that we don’t see regularly. I get so wrapped up in my own excitement to see my loved ones that I ... Read more

January 7, 2021January 7, 2021

FA Research Alliance Petitioning for Urgent Access to Omaveloxolone

News

With backing from a petition that is now circulating online, the Friedrich’s Ataxia Research Alliance (FARA) will ask Reata Pharmaceuticals and the U.S. Food and Drug Administration to make ... Read more

January 6, 2021January 4, 2021

COVID-19 Vaccines Bring Relief for Many, Envy for Some

Columns, Little Victories - a Column by Matthew Lafleur

Despite whatever successes we saw in 2020, the year mostly will be remembered for a pandemic that affected almost everyone on the planet. Though the death, financial hardships, joblessness, ... Read more