May 29, 2020May 29, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 28, 2020May 26, 2020
Instagram vs. Reality with Mobility Aids

Because of my Friedreich’s ataxia (FA), I use a walker as a mobility aid. The walker helps provide the stability that I can’t ... Read more
May 28, 2020May 28, 2020
Endurance Exercise Prevents Symptom Onset in FA Mouse Model, Study Finds

Endurance exercise prevented the onset of symptoms without the need to restore frataxin production in a mouse model of Friedreich’s ataxia (FA), a study ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 22, 2020May 22, 2020
Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, ... Read more

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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

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Endurance Exercise Prevents Symptom Onset in FA Mouse Model, Study Finds

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Investigational Gene Therapy AVXS-401 Shows Promise in Animal Models of FA

Learning to Shift from ‘I’ to ‘We’

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Pros and Cons of Mothering with Friedreich’s Ataxia

FARA Presenting Research Flash Talk Series During FA Awareness Month

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Playing the Cards We’ve Been Dealt

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

COVID-19 Delays Planned Trial of PTC743 and Gene Therapy Work, PTC Says

It’s OK to Ask Me About FA

Parallels Between 2018’s ‘Bird Box’ and the Coronavirus Pandemic

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

This Year’s rideATAXIA Was Postponed, but We’re Not Giving Up

Tiny RNA Molecules Involved in FA May Be Promising Treatment Candidates

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Choosing Our Own Identity

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Life Is Fragile, So Handle with Care

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Alterations in Nerve Cells’ Proteins May Lead to Neurodegeneration in FA, Mouse Study Suggests

The Zeego Tales: A Little Something Extra

Healthy Distractions and Keeping Busy

Training a New Puppy Isn’t Easy, But It’s Worth It

Eye Health Mirrors Neurologic Disability in FA Patients, Study Reports

Keeping a Homebound Physical Therapy Routine

Remembering What Matters During the Pandemic

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

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COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Instagram vs. Reality with Mobility Aids

Endurance Exercise Prevents Symptom Onset in FA Mouse Model, Study Finds

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Investigational Gene Therapy AVXS-401 Shows Promise in Animal Models of FA

Learning to Shift from ‘I’ to ‘We’

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Pros and Cons of Mothering with Friedreich’s Ataxia

FARA Presenting Research Flash Talk Series During FA Awareness Month

Subscribe to our mailing list

Playing the Cards We’ve Been Dealt

Remembering that Other People’s Opinions Matter

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Raise Awareness by Joining the ‘Lend Us Some Muscle’ Campaign

COVID-19 Delays Planned Trial of PTC743 and Gene Therapy Work, PTC Says

It’s OK to Ask Me About FA

Parallels Between 2018’s ‘Bird Box’ and the Coronavirus Pandemic

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

This Year’s rideATAXIA Was Postponed, but We’re Not Giving Up

Tiny RNA Molecules Involved in FA May Be Promising Treatment Candidates

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Choosing Our Own Identity

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Life Is Fragile, So Handle with Care

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Alterations in Nerve Cells’ Proteins May Lead to Neurodegeneration in FA, Mouse Study Suggests

The Zeego Tales: A Little Something Extra

Healthy Distractions and Keeping Busy

Training a New Puppy Isn’t Easy, But It’s Worth It

Eye Health Mirrors Neurologic Disability in FA Patients, Study Reports

Keeping a Homebound Physical Therapy Routine

Remembering What Matters During the Pandemic

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

Pin It on Pinterest