It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his…
Articles by Margarida Azevedo, MSc
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare…
People with rare diseases know that the right government policies can make a big difference in the quality of their…
The Friedreich’s Ataxia Research Alliance (FARA) and the University of California Los Angeles (UCLA) have joined forces to…
An attorney in Sioux Falls, South Dakota, has taken up the challenge to raise $75,000 before Christmas to fund a…
The European Medicines Agency (EMA) has granted orphan medicinal product designation to Agilis Biotherapeutics’ gene therapy candidate…
Haji Babar Naseer Sheikh was diagnosed with Friedreich’s ataxia (FA) soon after earning his bachelor’s degree. His worsening neurological…
Reata Pharmaceuticals has enrolled the first patient in the pivotal Part 2 of its MOXIe study designed to evaluate…
Omaveloxolone, a drug developed by Reata Pharmaceuticals, may slow disease progression and promote the recovery of lost neurological…
The Friedreich’s Ataxia Research Alliance is holding a fun and easy photo challenge to raise global awareness of the disease. Photography…