A substantial knowledge gap still exists about the prevalence of Friedreich’s ataxia (FA) around the world, as well as…
Articles by Marta Figueiredo, PhD
The National Ataxia Foundation (NAF) is accepting applications for funding of research projects on ataxia — a lack of…
Reata Pharmaceuticals has initiated a rolling submission of a new drug application seeking U.S. approval of its experimental oral therapy…
Design Therapeutics announced that it remains on track to launch the first clinical trial of its lead experimental candidate…
CRISPR Therapeutics has joined forces with Capsida Biotherapeutics to discover and develop new gene-editing therapies for Friedreich’s ataxia (FA) and…
Six months of treatment with interferon-gamma, known as IFN-gamma, is safe and leads to significant improvements in walking skills and…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
The Friedreich’s Ataxia Center of Excellence of the Children’s Hospital of Philadelphia (CHOP) has been awarded a total of…
Exicure recently discussed preclinical research data and the next steps in developing XCUR-FXN, its investigational antisense oligonucleotide (ASO) therapy…
PTC Therapeutics has launched a Phase 3 clinical trial to evaluate its investigational oral therapy vatiquinone (PTC743) in children…