Vatiquinone’s FDA rejection is the most recent event in a swathe of disappointing turnouts for the company, as the ...

The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid ...

PTC Therapeutics has long been trying to workshop its vatiquinone into a marketable treatment for Friedreich's ataxia (FA) ...

The U.S. Food and Drug Administration has denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...

The regulatory body determined that PTC Therapeutics’ New Drug Application for vatiquinone did not provide sufficient ...

FDA rejected PTC Therapeutics' vatiquinone for Friedreich's ataxia, citing insufficient efficacy data and requiring another ...

Today, the FDA issued a complete response letter (CRL) for vatiquinone (PTC Therapeutics), delaying its potential approval as a treatment for Friedreich ataxia amid efficacy concerns.

Friedreich’s ataxia is a rare disease, affecting an estimated 1 in 50,000 people in the United States. Without a strong network of support, caregiving can feel especially isolating.

Susan Perlman, MD, provides a comprehensive overview of Friedreich's ataxia management, encompassing current guidelines, FDA-approved therapies, assessment tools, and prospects for the treatment ...

A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia Lynch DR, Perlman SL, Meier T Arch Neurol. 2010;67:941-947 ...

The Friedreich’s ataxia locus (FRDA) maps on chromosome 9q13. Genetic data, obtained from a small number of recombination events, indicated that the FRDA locus might be located centromeric to ...