Stealth BioTherapeutics’ lead candidate elamipretide has been granted orphan drug status by the U.S. Food and Drug Administration (FDA)…
Articles by Patricia Inácio, PhD
The U.S. Food and Drug Administration (FDA) has granted its fast track designation to Reata Pharmaceuticals’ oral candidate…
A Phase 2a trial assessing Stealth BioTherapeutics‘ lead investigational product elamipretide in people with Friedreich’s ataxia (FA) is…
Daily dosing of CTI-1601, a lab-made, modified version of frataxin — the protein lacking in people with Friedreich’s…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
GeneDx has expanded its list of genetic tests to include the analysis of expansion repeats that underlie adult-onset ataxias,…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease and fast track designations to Retrotope’s investigational…
Lexeo Therapeutics recently launched with an initial funding of $85 million to develop gene therapies for Friedreich’s ataxia…
Eye health correlates with neurological disability in people with Friedreich’s ataxia (FA), a clinical study reports. These findings support the…