May 17, 2018May 17, 2018
Breath Testing Shows Promise as Reliable, Simple Method for Diagnosing FA, Other Diseases
News
Breath testing holds potential as an accurate, simple, and noninvasive diagnostic tool for Friedreich’s ataxia (FA) and other neurodegenerative diseases, ... Read more
May 17, 2018May 17, 2018
‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
News
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act ... Read more
May 17, 2018May 17, 2018
#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
News
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live ... Read more
May 15, 2018May 15, 2018
MDA Awards $2.6 Million in Grants to Improve Understanding of Neuromuscular Diseases
News
The Muscular Dystrophy Association (MDA) awarded 13 grants totaling $2.6 million to promote and support research projects aimed at better ... Read more
May 10, 2018May 10, 2018
Clinical Management of Adult Siblings with FA Described in Case Report
News
The clinical management of two siblings with Friedreich’s ataxia (FA) involved multiple approaches, including physical and occupational therapy, medications, orthopedic ... Read more
May 8, 2018May 8, 2018
Methylene Blue Analogues Improved Mitochondrial Function and Increased Frataxin Levels in Cell Study
News
Compounds similar to the chemical methylene blue, which has been shown to have therapeutic benefits in several diseases, were ... Read more
May 4, 2018May 4, 2018
#ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12
News
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ... Read more
May 3, 2018May 3, 2018
UK Pilot Study to Evaluate Effects of Stem Cell Therapy in Friedreich’s Ataxia Patients
News
The clinical benefits of stem cell therapy in Friedreich’s ataxia patients will be assessed in a small pilot study ... Read more
April 24, 2018April 24, 2018
FA Patients Show Gradual Worsening of Certain Auditory Responses, Study Says
News
Researchers have found that patients with Friedreich’s ataxia (FA) show progressive deterioration of auditory neural responses to ongoing ... Read more
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 ... 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. ... 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 ... Read more

May 17, 2018May 17, 2018

Breath Testing Shows Promise as Reliable, Simple Method for Diagnosing FA, Other Diseases

News

Breath testing holds potential as an accurate, simple, and noninvasive diagnostic tool for Friedreich’s ataxia (FA) and other neurodegenerative diseases, a review suggests. The study, “Emergence of breath testing as ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 17, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 15, 2018May 15, 2018

MDA Awards $2.6 Million in Grants to Improve Understanding of Neuromuscular Diseases

News

The Muscular Dystrophy Association (MDA) awarded 13 grants totaling $2.6 million to promote and support research projects aimed at better understanding and treating muscular dystrophies and related life-threatening diseases, including ... Read more

May 10, 2018May 10, 2018

Clinical Management of Adult Siblings with FA Described in Case Report

News

The clinical management of two siblings with Friedreich’s ataxia (FA) involved multiple approaches, including physical and occupational therapy, medications, orthopedic devices, and nutritional supplements, according to a report from clinicians ... Read more

May 8, 2018May 8, 2018

Methylene Blue Analogues Improved Mitochondrial Function and Increased Frataxin Levels in Cell Study

News

Compounds similar to the chemical methylene blue, which has been shown to have therapeutic benefits in several diseases, were seen to increase frataxin levels and the production of mitochondria ... Read more

May 4, 2018May 4, 2018

#ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan ... Read more

May 3, 2018May 3, 2018

UK Pilot Study to Evaluate Effects of Stem Cell Therapy in Friedreich’s Ataxia Patients

News

The clinical benefits of stem cell therapy in Friedreich’s ataxia patients will be assessed in a small pilot study at the University of Bristol in the United Kingdom. Friedreich’s ataxia (FA) ... Read more

April 24, 2018April 24, 2018

FA Patients Show Gradual Worsening of Certain Auditory Responses, Study Says

News

Researchers have found that patients with Friedreich’s ataxia (FA) show progressive deterioration of auditory neural responses to ongoing acoustic stimulation. The study titled “Rapid exhaustion of auditory neural conduction ... Read more

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